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ANNUAL REPORTSharing Knowledge. Sharing Hope.

2018 Annual Report

January – December Milwaukee Campus Medical College of Wisconsin

9200 W Wisconsin Ave, Suite C5500 Milwaukee, WI 53226 (414) 805-0700Minneapolis Campus National Marrow Donor

Program/ Be The Match 500 N 5th St Minneapolis, MN 55401 (763) 406-5800

cibmtr.org

CIBMTR 2018 Annual Report TABLE OF CONTENTS

TABLE OF CONTENTS

1.0 WHO WE ARE ........................................................................................................................................ 1

1.1 Mission .............................................................................................................................................. 1

1.2 Value to the Community ................................................................................................................... 1

1.3 Organizational Structure ................................................................................................................... 2

1.3.1 Scientific Working Committees .................................................................................................. 2

2.0 WHAT WE DO ........................................................................................................................................ 7

2.1 Clinical Outcomes Research Program ............................................................................................. 12

2.1.1 Scientific Working Committees ................................................................................................ 12

2.1.2 Stem Cell Therapeutic Outcomes Database ............................................................................. 16

2.1.3 Cellular Therapy Research Initiatives ....................................................................................... 17

2.1.4 CMS Coverage with Evidence Development Studies ............................................................... 19

2.1.5 Patient-Reported Outcomes .................................................................................................... 20

2.1.6 International Initiatives ............................................................................................................ 20

2.2 Immunobiology Research Program ................................................................................................. 22

2.3 Clinical Trials Support Program ....................................................................................................... 24

2.3.1 Blood and Marrow Transplant Clinical Trials Network ............................................................ 24

2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation ............................ 26

2.4 Health Services Research Program.................................................................................................. 28

2.5 Bioinformatics Research Program ................................................................................................... 30

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2.6 Statistical Methodology Research Program .................................................................................... 31

2.7 Corporate Program .......................................................................................................................... 32

3.0 HOW WE SHARE KNOWLEDGE ........................................................................................................... 33

3.1 Information Request Service ........................................................................................................... 38

3.2 Internet Presence ............................................................................................................................ 39

3.2.1 CIBMTR Public Website ............................................................................................................ 39

3.2.2 CIBMTR Collaborative Site ........................................................................................................ 40

3.2.3 CIBMTR Portal Site .................................................................................................................... 40

3.2.4 Be The Match Public Website................................................................................................... 42

3.2.5 Be The Match Clinical Website ................................................................................................. 42

3.2.6 HRSA CWBYCTP Website .......................................................................................................... 43

3.2.7 Other Applications and Data Exchange Standards ................................................................... 43

3.3 Annual Meetings ............................................................................................................................. 45

3.3.1 BMT Tandem / TCT Meetings ................................................................................................... 45

3.3.2 Cellular Therapy Registry Forum .............................................................................................. 46

3.4 Data Management Training ............................................................................................................ 47

4.0 HOW WE COLLECT AND MANAGE DATA ............................................................................................ 48

4.1 Research Data Life Cycle ................................................................................................................. 48 4.2 Collecting and Storing Data ............................................................................................................. 49

4.2.1 FormsNet .................................................................................................................................. 49

4.2.2 Research Database ...................................................................................................................

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49

4.2.3 Integrated Data Warehouse ..................................................................................................... 49

4.3 Ensuring Data Quality ...................................................................................................................... 50

4.3.1 Continuous Process Improvement ........................................................................................... 50

4.3.2 Verification and Validation ....................................................................................................... 50

4.3.3 On-Site Data Audit Program ..................................................................................................... 51

4.4 Protecting Patients and Data .......................................................................................................... 53

4.4.1 Human Subjects / HIPAA Compliance ...................................................................................... 53

4.4.2 Information Security and Data Privacy ..................................................................................... 53

5.0 WHAT WE WILL DO NEXT.................................................................................................................... 54

2018 KEY ACCOMPLISHMENTS ................................................................................................................. 57

APPENDIX A: CENTERS .............................................................................................................................. 61

Appendix A1: US Centers....................................................................................................................... 61

Appendix A2: International Centers ...................................................................................................... 72

APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP ..................... 82 Appendix B1: Organizational Structure – Milwaukee Campus ............................................................. 83

Appendix B2: Organizational Structure – Minneapolis Campus ........................................................... 85

Appendix B3: Coordinating Center Leadership ..................................................................................... 88

APPENDIX C: COMMITTEE MEMBERSHIP ................................................................................................. 98

Appendix C1: Advisory Committee Membership .................................................................................. 98

Appendix C2: Executive Committee Membership .............................................................................. 101

Appendix C3: Consumer Advocacy Committee Membership .............................................................

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103

Appendix C4: Nominating Committee Membership ........................................................................... 104

Appendix C5: Scientific Working Committee Leadership ................................................................... 105

Appendix C6: Immunobiology Steering Committee Membership ...................................................... 109

Appendix C7: Clinical Trials Advisory Committee Membership .......................................................... 110

APPENDIX D: PUBLICATIONS ................................................................................................................... 111

Appendix D1: Scientific Working Committee Publications ................................................................. 111

Appendix D2: BMT CTN Publications .................................................................................................. 128

Appendix D3: RCI BMT Publications .................................................................................................... 131

Appendix D4: Health Services Research Program Publications .......................................................... 132

Appendix D5: Bioinformatics Research Program Publications ........................................................... 134

Appendix D6: Statistical Methodology Research Program Publications ............................................ 136

Appendix D7: 2017 Publications Not Previously Reported ................................................................. 137

APPENDIX E: PRESENTATIONS ................................................................................................................ 138

APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ..................................................... 146 APPENDIX G: CLINICAL STUDIES AND TRIALS ......................................................................................... 149

Appendix G1: BMT CTN Clinical Trials Open for Enrollment ............................................................... 149Appendix G2: RCI BMT Clinical studies ............................................................................................... 151

APPENDIX H: FORMS SUBMISSION PROCESS ......................................................................................... 154

APPENDIX I: WEBSITES ............................................................................................................................ 155

APPENDIX J: GLOSSARY ........................................................................................................................... 156

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FIGURES AND TABLES Figure 1.1 CIBMTR Research Programs....................................................................................................... 1

Figure 1.2 Scientific Organizational Structure ............................................................................................ 3

Figure 1.3 Functional Organizational Structure with Scientific Oversight .................................................. 4

Table 1.4 Committee Structure .................................................................................................................. 5

Figure 2.1 Continued Growth in the Number of Patients Registered with the CIBMTR ............................ 8

Figure and Table 2.2 Distribution of Patients in the CIBMTR Research Database by Graft Source ........... 8 Table 2.3 Distribution of Transplant Patients in the CIBMTR Research Database by Indication ............... 9 Table 2.4 2018 CIBMTR Publications by Journal ....................................................................................... 10

Figure 2.5 CIBMTR Publications by Year ................................................................................................... 10

Table 2.6 2018 CIBMTR Presentations by Meeting .................................................................................. 11

Table 2.7 2018 Working Committee Studies ............................................................................................ 13

Figure 2.8 2018 CIBMTR Publications by Program ................................................................................... 14

Figure 2.9 Working Committee Study Proposal Review Process.............................................................. 15

Table 2.10 CMS CED Studies ..................................................................................................................... 19

Figure 3.1 How to Access CIBMTR Knowledge ......................................................................................... 33

Table 3.2 How to Access Information Online ........................................................................................... 34

Table 3.3 How to Access Data Online ....................................................................................................... 34

Table 3.4 How to Access Tools Online ...................................................................................................... 35

Table 3.5 How to Access Biospecimens Online ........................................................................................ 35

Table 3.6 Standard Reports Published by the CIBMTR ............................................................................. 36

Table 3.7 Data Requests Addressed by the CIBMTR in 2018.................................................................... 38

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Figure 4.1 Research Data Life Cycle .......................................................................................................... 48

Figure 4.2 Audit Process ........................................................................................................................... 52

Table 5.1 Plans to Enhance Data ............................................................................................................... 54

Table 5.2 Plans to Expand Knowledge Sharing ......................................................................................... 55

Table 5.3 Plans to Increase Impact ........................................................................................................... 56

WEB LINKS Throughout this report, electronic links to webpages and documents are provided; they are underlined and italicized for identification. If you are unable to access items using the links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).

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CIBMTR 2018 Annual Report

1.0 WHO WE ARE

1.0 WHO WE ARE

The CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW).

1.1 MISSION The CIBMTR promotes collaborative research to understand and improve access to and outcomes of cellular therapies for the people we serve.

1.2 VALUE TO THE COMMUNITY The CIBMTR has collected health outcomes data

worldwide for >45 years, resulting in a Research

Database with information on >500,000 patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of participating centers, an extensive clinical outcomes database, and a unique

biospecimen repository. CIBMTR research involves 6 major programs (Figure 1.1).

Figure 1.1 CIBMTR Research Programs

Clinical Outcomes

Immunobiology

Clinical Trials

Health Services

Bioinformatics

Statistical Methodology

15 Scientific Working Committees utilize the CIBMTR’s Research Database to answer clinically important questions. Each committee focuses on a specific disease or condition, use of cellular therapies, or complication of treatment.

The CIBMTR manages a repository of paired tissue samples from donors and recipients, both unrelated and related, to study the genetic, cellular, and immunologic factors that influence transplant outcomes.

The BMT CTN conducts multicenter Phase II and III trials with broad national participation while the RCI BMT supports smaller trials that bridge the gap between single-center studies and larger trials.

The CIBMTR explores how social factors, financial systems, care processes, and behavior affect access to and outcomes of cellular therapies. Current studies not only improve practice but also address barriers to treatment.

The CIBMTR provides expertise in, and conducts research on, translational and operational bioinformatics. Recent endeavors include improving the HLA matching algorithm and analyzing next generation sequencing typing data.

The CIBMTR Coordinating Center provides advice and statistical consultation to researchers developing protocols for cellular therapy studies and investigates new statistical approaches


and techniques for analyzing cellular therapy data.Page | 1

1.3 ORGANIZATIONAL STRUCTURE The CIBMTR (Figures 1.2 and 1.3) represents a network of >400 participating centers in >40 countries (Appendix A) that submit outcomesrelated data for patients. The CIBMTR Coordinating Center, staffed by approximately 185 employees (Appendix B), provides data acquisition, management, and statistical support for analyses of these data.

The Chief Scientific Director is responsible for all administrative and scientific operations. The CIBMTR utilizes a dyad leadership structure, which partners administrative management with Senior Scientific Directors who provide scientific input to each functional area of the Coordinating Center (Figure 1.3). CIBMTR Administrative Committees (Table 1.4) provide input and advice to the internal leadership team, ensuring the continued support of the needs and priorities of the scientific and medical communities.

1.3.1 Scientific Working Committees

To ensure broad input into the research process and efficient use of resources, the

CIBMTR looks to 15 Scientific Working Committees focused on specific research areas.

Total Working Committee membership exceeds 2,700 researchers. Membership is open to anyone interested in participating. Many members are clinical researchers, but statisticians, basic scientists, patients, caregivers, and others participate in developing and conducting studies that use CIBMTR data and / or resources. PhD-level statistical faculty and Master’s-level statisticians from the CIBMTR Coordinating Center provide unique expertise in data analysis. Basic scientists

investigating human leukocyte antigen (HLA), immunogenetics, pharmacogenetics, stem cell biology, and other areas related to cellular therapy provide essential expertise in their respective areas.

1.0 WHO WE ARE

Scientific Working Committees Acute Leukemia

Autoimmune Diseases and Cellular Therapies

Chronic Leukemia

Donor Health and Safety

Graft Sources and Manipulation

Graft-versus-Host Disease

Health Services and International Studies

Immunobiology

Infection and Immune Reconstitution

Late Effects and Quality of Life

Lymphoma

Pediatric Cancer

Plasma Cell Disorders and Adult Solid Tumors

Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders

Regimen-Related Toxicity and Supportive Care

The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities.


Working Committee Leadership Chairs (usually 3-4)

MD Scientific Director

PhD Statistical Director

MS-level Statistician

Working Committee leadership is listed in Appendix C5.

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CIBMTR 2018 Annual Report 1.0 WHO WE ARE

CIBMTR 2018 Annual Report Figure 1.2 Scientific Organizational Structure

K Flynn, PhDOutcomes

Reported -for PatientSenior Scientific Director

M Maiers, MSResearch Program

Bioinfomatics

Advisory role

B Shaw, MD, PhDfor Data Operations

Senior Scientific Director Advisory role

B Shaw, MD, PhDL Burns, MD

S Devine, MD(RCI BMT)

Blood and Marrow TransplantationResource for Clinical Investigations in

M Pasquini, MD, MS(BMT CTN)

Trials NetworkBlood and Marrow Transplant Clinical

B Shaw, MD, PhDM Pasquini, MD, MS

Support ProgramClinical Trials

S Lee, MD, MPHResearch Program

Immunobiology

L Burns, MDResearch Program

Health Services

M Eapen, MBBS, MSClinical Outcomes

MJ Zhang, PhDResearch Program

Methodology Statistical

MJ Zhang, PhDDirector

Chief Statistical

CommitteeConsumer Advocacy

Steering CommitteesScientific Working Committees

M Horowitz, MD, MSChief Scientific Director

S Devine, MDMinneapolis

Director for CIBMTR Associate Scientific

JD Rizzo, MD, MSfor SCTOD

Senior Scientific Director

M Eapen, MBBS, MSfor Research OperationsSenior Scientific Director

D Weisdorf, MDSenior Research Advisor

WisconsinMedical College of

Executive Committee

CommitteeAdvisory

AssemblyCIBMTR

Transplant Centers Program / Be The MatchNational Marrow Donor

CR Mills, PhDExecutive Director

Joint Affiliation Committee

CIBMTR 2018 Annual Report Page | 3

1.0 WHO WE ARE

CIBMTR 2018 Annual Report Figure 1.3 Functional Organizational Structure with Scientific Oversight


Page | 4

CIBMTR 2018 Annual Report 1.0 WHO WE ARE

Table 1.4 Committee Structure Committee Function Meetings Roster

Joint Affiliation Board

• Reviews and approves the CIBMTR budget and research plan

• Amends the terms of the affiliation agreement, as necessary

• Reviews and approves data access and confidentiality policies

• Annually

Assembly • Includes representatives from each center

that submits CRF-level data • Elects members of the Advisory,

Nominating, and Clinical Trials Advisory Committees

• Annually during the TCT | Transplantation & Cellular

Therapy Meetings of ASBMT and CIBMTR

(TCT Meetings)

Advisory Committee • Oversees CIBMTR policies and scientific agenda

• Partners with the Working Committees to prioritize scientific studies

• Oversees operations of the SCTOD

• In person annually at the TCT Meetings

• By teleconference quarterly and as needed

Appendix C1

Executive Committee (subcommittee of Advisory Committee)

• Provides scientific and policy advice to the Chief Scientific Director and Coordinating Center

• Reviews audit results and makes recommendations for improvement

• Four times annually by teleconference

Appendix C2

Consumer Advocacy Committee (subcommittee of Advisory Committee)

• Provides patient and donor perspectives during the development of the CIBMTR research agenda

• Communicates CIBMTR research results and data to the non-medical community


• By teleconference periodically

Appendix C3

Page | 5

CIBMTR 2018 Annual Report Nominating Committee

• Prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees

• Makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments

• At least once annually each Fall by teleconference

Appendix C4

1.0 WHO WE ARE Committee Function Meetings Roster

Scientific Working Committees (Section 1.3.1)

• Design and conduct relevant studies using CIBMTR data,

statistical resources, networks, and / or centers

• Set priorities for clinical outcomes studies • Assess and revise CIBMTR data collection

forms, as needed


• Leadership - by teleconference every 4-8 weeks

Leadership - Appendix C5

Immunobiology Steering Committee / NMDP/Be The Match Histocompatibility Advisory Group

• Reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies

• In person twice annually, in summer and at the TCT Meetings

Appendix C6

Clinical Trials • Makes recommendations regarding • In person annually at Appendix C7 Steering the RCI BMT’s strategy, direction, the TCT Meetings Committee and alignment with the CIBMTR’s • By teleconference as

scientific agenda needed

2.0 WHAT WE DO

The CIBMTR collects data for approximately 25,000 new patients annually as well as a continually increasing volume of follow-up

data on previously reported recipients and donors. Centers submit transplant data at two levels: A Transplant Essential Data (TED) level,

which captures basic data, and a Comprehensive Report Form (CRF) level, which captures more detail. Centers submit cellular therapy data (Section 2.1.3) with a suite of Cellular Therapy Essential Data (CTED) forms.

Submission of outcomes data is mandatory for allogeneic transplants in the United States (US)

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CIBMTR 2018 Annual Report 2.0 WHAT WE DO and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and >80% of US autologous transplants are reported. Cellular therapy outcomes data are also reported to the CIBMTR; since July 2016, >2,000 patients who received cellular therapy were included in the CIBMTR Research Database. Data for approximately 7,500 non-US patients are collected annually.

The CIBMTR Research Database contains information on >500,000 patients. Figure 2.1 shows the continued growth in the number of patients registered with the CIBMTR. The distribution of transplant patients in the database by graft type is displayed in Figure and Table 2.2 and by disease in Table 2.3. Research

The CIBMTR is dedicated to improving survival, treatment, and quality of life for cellular therapy patients. We provide many opportunities to conduct research utilizing CIBMTR resources, and we encourage both senior and junior investigators to participate.

At any given time, the CIBMTR has >200 retrospective, correlative, or methodologic studies and 15 prospective trials ongoing in 6 major areas of research activity.

Areas of Research Activity Clinical Outcomes Research (Section 2.1)

Immunobiology Research (Section 2.2)

Clinical Trials Support (Section 2.3)

• Blood and Marrow Transplant Clinical Trials Network (BMT CTN) (Section 2.3.1)

• Resource for Clinical Investigations in Blood and Marrow Transplantation (RCI BMT)

(Section 2.3.2)

Health Services Research (Section 2.4)

Bioinformatics Research (Section 2.5)

Statistical Methodology Research (Section 2.6)

Publications

In 2018, the CIBMTR published 109 peerreviewed manuscripts in the journals listed in Table 2.4. As of December 31, an additional 31 manuscripts were submitted and are under review, and 2 manuscripts are in press. A complete list of 2018 publications is provided in Appendix D. Figure 2.5 displays the number of CIBMTR publications annually since 2004. Presentations

In 2018, CIBMTR study investigators presented 76 abstracts (47 oral and 29 poster) at national and international conferences (Table 2.6). A complete list of presentations is provided in Appendix E.

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CIBMTR 2018 Annual Report 2.0 WHAT WE DO

Figure 2.1 Continued Growth in the Number of Patients Registered with the CIBMTR

Years (Data are incomplete for 2018)

Figure and Table 2.2 Distribution of Transplant Patients in the CIBMTR Research Database by Graft Source

Allogeneic Autologous TOTAL

TED CRF TED CRF

Bone Marrow 53,832 60,889 10,158 5,911 130,790

Peripheral Blood 84,844 38,065 193,932 40,799 357,640

Cord Blood 5,903 9,737 29 7 15,676

TOTAL 144,579 108,691 204,119 46,717 504,106

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Table 2.3 Distribution of Transplant Patients in the CIBMTR Research Database by Indication

TRANSPLANT DATA Allogeneic Autologous TOTAL

Indication TED CRF TED CRF

Lymphoma 13,518 6,967 78,962 15,193 114,640

Plasma cell disorders 2,701 1,468 83,375 15,575 103,119

Acute myelogenous leukemia 49,119 30,642 6,210 2,442 88,413

Other malignant diseases1 6,618 4,008 32,260 12,473 55,359

Acute lymphoblastic leukemia 25,090 17,931 1,193 477 44,691

Chronic myelogenous leukemia 14,621 15,306 420 287 30,634

Myelodysplastic / myeloproliferative syndromes 15,818 13,305 221 91 29,435

Aplastic anemia / PNH2 7,060 7,874 - - 14,934

Immune disorders3 3,864 4,222 - - 8,086

Hemoglobinopathy4 3,021 3,350 - - 6,371

Inherited bone marrow failure5 1,402 1,744 - - 3,146

Inborn errors of metabolism 1,196 1,608 - - 2,804

Other nonmalignant disorders6 193 164 1,036 163 1,556

Other diseases 358 102 442 16 918

TOTAL 144,579 108,691 204,119 46,717 504,106

1. Includes other leukemia (9,217 allogeneic and 981 autologous) and solid tumors (1,409 allogeneic and 43,752 autologous)

2. Includes severe aplastic anemia (14,284 allogeneic) and paroxysmal nocturnal hemoglobinuria (650 allogeneic)

3. Includes immune deficiencies (6,368 allogeneic) and histiocytic disorders (1,718 allogeneic) 4. Includes sickle cell anemia (1,911 allogeneic), sickle cell thalassemia (153 allogeneic), and

thalassemia major (4,307 allogeneic) 5. Includes Schwachmann-Diamond (78 allogeneic), Fanconi anemia (2,276 allogeneic),

Diamond-Blackfan anemia (437 allogeneic), and other inherited abnormalities of erythrocyte (355 allogeneic)

6. Includes platelet disorders (215 allogeneic and 7 autologous) and autoimmune deficiencies (142 allogeneic and 1,192 autologous)

Table 2.4 2018 CIBMTR Publications by Journal

Journal Number of Publications

Biology of Blood and Marrow Transplantation 38

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Bone Marrow Transplantation 16

Blood Advances 11

Haematologica 7

Blood 6

Cancer 4

Human Immunology 3

Journal of Allergy and Clinical Immunology 2

Other Journals* 22

TOTAL 109

*One publication each in the American Journal of Hematology, Biometrical Journal, Biometrics, British Journal of Haematology, Cells, Frontiers in Immunology, Genes and Immunity, HLA, Immunogenetics, Infection Control and Hospital Epidemiology, Journal of Cancer Education, Journal of Clinical Oncology, Journal of Pediatric Hematology/Oncology, Journal of Thoracic Oncology, Lancet Haematology, Leukemia Research, Lifetime Data Analysis, Molecular Biology Reports, Palliative and Supportive Care, PLOS One, Scientific Reports, and Statistics in Medicine.

Figure 2.5 CIBMTR Publications by Year

Page | 10


Table 2.6 2018 CIBMTR Presentations by Meeting Conference Oral Poster Total

American Society of Hematology (ASH) 14 10 24

BMT Tandem Meetings 12 7 18

European Society for Blood and Marrow Transplantation (EBMT) 3 3 6

Data Standards and Symposium Hackathon 6 0 6

American Society of Histocompatibility and Immunogenetics 2 3 5

European Federation for Immunogenetics 1 2 3

American Society of Clinical Oncology 0 2 2

Eastern North American Region International Biometric Society 2 0 2

NMDP/Be The Match Council Meeting 2 0 2

Other Meetings and Conferences* 5 2 7

TOTAL 47 29 76

*One oral presentation each at the American Society of Pediatric Hematology/Oncology Annual Conference, HLA and KIR Population Dynamics Workshop, Joint Statistical Meetings of the AmericanStatistical Association, KIR Workshop, and National Sickle Cell Disease Association of America Annual Meeting. One poster presentation each at the Academy Health Annual Research Meeting and Canadian Blood and Marrow Transplant Group Annual Meeting.

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2.1 CLINICAL OUTCOMES RESEARCH PROGRAMClinical Outcomes Research using the CIBMTR Research Database is a core activity of the organization. These studies address a wide range of issues, focusing on questions that are difficult or impossible to address in singlecenter studies or randomized trials because diseases studied are uncommon, single centers treat few patients with a given disorder, and not all important questions are amenable to a randomized research design.

2.1.1 Scientific Working Committees

Program Activities

The 15 Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research with approximately 200 studies in progress. Numbers of studies in progress, publications, and presentations by each Working Committee are displayed in Table 2.7. In progress and recently published studies are

detailed in the 2018 Working Committee Research Portfolio on the CIBMTR website.

The Working Committees reviewed 205 new study proposals before the 2018 BMT Tandem Meetings. 98 proposals were presented at the annual meeting, and 41 were approved. The prioritization and selection process ensures that the most important issues can be addressed in a timely manner. Publications

In 2018, Working Committee study investigators published 74 peer-reviewed manuscripts in medical journals. This is >65% of the total number of CIBMTR publications this year (Figure 2.8). A complete list of Working Committee publications is provided in Appendix D1.

Presentations

In 2018, Working Committee study

Page | 12


investigators presented 46 abstracts (28 oral Key Working Committee

Publications this Year

Buchbinder D, Kelly DL, Duarte RF, et al. Neurocognitive dysfunction in HCT recipients: Expert review from the CIBMTR and EBMT. Bone Marrow Transplantation. 2018 May 1; 53(5): 535555. Epub 2018 Jan 17. PMC5985976.

Smith SM, Godfrey J, Ahn KW, et al. Autologous HCT versus allogeneic HCT in patients with follicular lymphoma experiencing early treatment failure. Cancer. 2018 Jun 15; 124(12): 2541-2551. Epub 2018 Apr 12. PMC5990449.

Nikiforow S, Wang T, Hemmer M, et al. Upper gastrointestinal acute GVHD adds minimal prognostic value in isolation or with other GVHD symptoms as currently diagnosed and treated. Haematologica.

2018 Oct 1; 103(10): 1708-1719. Epub 2018 Aug 3. PMC6165812.

Keesler DA, Martin AS, Bonfim C, et al. Bone marrow versus peripheral blood from unrelated donors for children and adolescents with acute leukemia. Biology of Blood and Marrow Transplantation. 2018 Dec 1; 24(12): 2487-2492. Epub 2018 Aug 21. PMC6286246.

Marsh R, Hebert KM, Keesler D, et al. Practice pattern changes and improvements in HCT for primary immunodeficiencies. Journal of Allergy and Clinical Immunology. 2018 Dec 1; 142(6): 2004-2007. Epub 2018 Aug 28. PMC6289686.

and 18 poster). A complete list of CIBMTR presentations is provided in Appendix E.

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Table 2.7 2018 Working Committee Studies

Page | 14

Working Committee Studies in Progress

Publications Presentations

Acute Leukemia 13 2 (poster)

Autoimmune Diseases and Cellular Therapies 7 2 0

Chronic Leukemia 14 1 (3 oral & 1 poster)

Donor Health and Safety 14 4 (4 oral & 2 poster)

Graft Sources and Manipulation 7 5 (oral)

Graft-versus-Host Disease 13 8 (poster)

Health Services and International Studies 11 3 (1 oral & 2 poster)

Immunobiology 34 19 (6 oral & 2 poster)

Infection and Immune Reconstitution 9 1 (poster)

Late Effects and Quality of Life 13 12 (oral)

Lymphoma 9 3 (oral)

Pediatric Cancer 2 1 0

Plasma Cell Disorders and Adult Solid Tumors 10 3 0

Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders

12 6 (oral)

Regimen-Related Toxicity and Supportive Care 27 4 (6 oral & 1 poster)

TOTAL 195 74 46 (28 oral & 18 poster)


Funding

Support for the Working Committees is primarily provided by the National Institutes of Health (NIH) grant # U24CA076518 from the National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute for Allergy and Infectious Disease (NIAID).

How to Get Involved

Working Committees are collaborative in nature, and all interested individuals are encouraged to participate. Please feel free to attend annual in-person meetings of the Working Committees at the TCT Meetings in February. Additionally, anyone willing to follow the study development and management process (Appendix F) is eligible to propose a study to the Working Committees (Figure 2.9).

Figure 2.8 2018 CIBMTR Publications by Program

Page | 15

Successful Working Committee Study Proposals are

Feasible. Utilize data available in the CIBMTR Research Database.

Unique. Fill a gap not addressed by current studies or publications.

Important. Impact the field by improving cellular therapy procedures or results.

See the CIBMTR How to Propose a Study webpage and, specifically, the Study Proposal Outline on that webpage for additional guidelines and advice.


Figure 2.9 Working Committee Study Proposal Review Process

Page | 16


Page | 17

approval / rejection by the end of April.Working Committee Leadership contacts study investigator to notify of study•

Notification

proposals to pursue. Advisory Committee approves the CIBMTR research agenda.Working Committee Leadership utilizes member feedback in determining which•

ApprovalFinal

score to each.Working Committee members vote for each proposal, assigning a scientific impact•

Voting

the February TCT Meetings.Study investigator presents the proposal at the Working Committee meeting at•

Presentation

characteristics of patient data based on the population defined in the proposal.MS-level Statistician contacts the study investigator and prepares a table ofIf Working Committee Leadership clears the proposal to move forward, the•

AssessmentPreliminary

their proposals.timely fashion. Researchers with similar concepts may be advised to combineconflict with active studies, scientific merit, and ability to complete the study in aWorking Committee Leadership reviews for feasibility with CIBMTR data, potential•

ReviewInitial

Coordinating Center for consideration at the next TCT Meetings.By mid-November, study investigator submits proposal to the CIBMTR•

Submission


2.1.2 Stem Cell Therapeutic Outcomes

Database (SCTOD)

The CIBMTR administers the SCTOD contract for the Health Resources and Services Administration (HRSA)-sponsored C.W. Bill Young Cell Transplantation Program (CWBYCTP), established by the Stem Cell Therapeutic and Research Act of 2005 and renewed through the Stem Cell Therapeutic and Research Reauthorization Acts of 2010 and 2015.

C.W. Bill Young Cell Transplantation Program Goals

Collect, analyze, and report outcomes data for all allogeneic transplants and other therapeutic uses of blood stem cells

Publicize information about HCT to patients, families, health care professionals, and the public

Define better processes for identifying unrelated matched marrow donors, peripheral blood stem cell (PBSC) donors, and cord blood units through one electronic system

Increase availability of unrelated adult volunteer donors and cord blood units

Expand research to improve patient outcomes

Program Activities

For the SCTOD, the CIBMTR tracks and analyzes data for all allogeneic transplants performed in the US and transplants performed globally with products from the US. Annually, the CIBMTR publishes HCT volumes and performance data by transplant

Page | 18


center and provides public access to this information via the HRSA Blood Cell Transpla nt website. Center-Specific Volumes and Survival Analysis. As part of the contract to operate the SCTOD, the CIBMTR provides the annual volume of transplants performed at each center and performs a center-specific survival analysis evaluating the one-year survival rates among US centers. The report assesses transplants from both related and unrelated donors.

The most recent analysis was completed in September 2018 and contains information on all first allogeneic transplants performed in US centers from January 1, 2014, through December 31, 2016.

Center Outcomes Forums. The CIBMTR has conducted 6 Center Outcomes Forums to engage relevant stakeholders in the centerspecific outcomes reporting process.

The most recent meeting was held in September 2018. Recommendations were generated related to the center-specific analysis modeling, risk adjustment for pediatric non-malignant disease, statistical methodology, and improving collaboration to achieve quality improvement.

Cellular Therapies. The CWBYCTP also provides for collection of data for use of cells found in bone marrow, peripheral blood, and umbilical cord blood for alternative therapeutic applications. The CIBMTR captures uses of cells for the treatment of diseases without the intention of replacing the recipient’s hematopoietic function. The CIBMTR Cellular Therapy Outcomes Registry initiative (Section 2.1.3) supports detailed data collection of cellular therapies.

Study Summaries for Patients. In conjunction with NMDP/Be the Match and the Consumer Advocacy Committee, the CIBMTR publishes lay summaries of CIBMTR publications for

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patients and their loved ones. In 2018, the CIBMTR published 23 patient-friendly research summaries.

Funding

Support for the SCTOD is provided by the HRSA contract # HHSH250201700006C. How to Get Involved

All US centers performing allogeneic HCTs provide data to the CIBMTR for the SCTOD. These data are used to generate reports, which are distributed to transplant center medical directors and posted on the HRSA CWBYCTP website.

Publicly Available Reports developed by the CIBMTR for the SCTOD

Transplant Outcomes and Data

• US Patient Survival Report

• US Transplant Data by Center Report

• US Transplant Data by Disease Report

• Transplant Activity Report

These reports are available on the HRSA CWBYCTP website.

2.1.3 Cellular Therapy Research Initiatives

Program Activities

In addition to receiving data on transplant patients, the CIBMTR received data from >200 centers for >2,000 patients who received cellular therapy. The CIBMTR receives these data via a suite of CTED forms. The CIBMTR continues to work with international registries to review and harmonize data collection globally.

Since 2016, the CIBMTR has collected data for >500 patients who received chimeric antigen receptor (CAR) T cell therapy. >300 patients

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were treated for non-Hodgkin lymphoma; >175 were treated for acute lymphocytic leukemia and multiple myeloma.

As of December 2018, the CIBMTR Research Database includes >200 patients who received regenerative medicine. Patients were treated for neurologic disorders, cardiovascular diseases, immune deficiencies, and inherited disorders of metabolism.

Cellular Therapy Registry Forum. In October 2018, the CIBMTR held a fourth Cellular Therapy Registry Forum (Section 3.3.2). Topics included CAR T toxicity grading criteria and reporting, cellular therapy center accreditation and data auditing, long-term follow-up infrastructure, considerations for capturing biospecimens in cellular therapy recipients, and updates from federal agencies.

Long-Term Follow-Up. The Food and Drug Administration (FDA) requires pharmaceutical companies that commercialize genetically

engineered cellular therapies to follow recipients of these therapies for 15 years in order to evaluate their safety and efficacy. The CIBMTR can support this requirement and is currently partnered with several pharmaceutical companies to track these longterm outcome data.

Purpose of the Cellular Immunotherapy Data Resource

(CIDR)

Support the Immuno-Oncology Transplantation Network (IOTN)

Provide an infrastructure to manage data collection and verification from cellular therapies across multiple sources

Facilitate retrospective observational research

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Cellular Immunotherapy Data Resource. The CIBMTR received funding this year from the NIH to serve as the CIDR to collect outcomes of patients receiving cellular immunotherapies to support observational studies and inform prospective studies and clinical trials. The IOTN supports the Cancer MoonshotSM initiative to accelerate cancer research to make more therapies available to more patients.

In October 2018, the CIBMTR held a CIDR overview meeting to share information about the initiative and solicit input regarding the CIDR’s structure. The CIBMTR established an interim CIDR Executive Committee to provide scientific and policy advice and is in the process of establishing the CIDR Oversight Committee to provide input to the CIDR Executive Committee. Regenerative Medicine Outcomes Registry Strategy Meeting. The CIBMTR hosted a second regenerative medicine outcomes

registry strategy meeting in September 2018 in association with Cord Blood Connect, an international conference hosted by the Cord Blood Association. This meeting focused not only on sharing the CIBMTR’s registry capabilities, including form development and collection of patient-reported outcomes, but also on determining disease priority and establishing working groups related to the regenerative medicine outcomes registry. Four regenerative medicine indications were selected by the group to constitute the pilot data for the registry. Funding

Support for the CIDR is provided by NIH grant # U24CA233032 from the NCI and National Institute on Minority Health and Health Disparities. Support for other cellular therapy research initiatives is provided by NIH grant # U24CA076518, HRSA contract # HHSH250201700006C, and Office of Naval Research grant # N00014-17-1-2850.

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How to Get Involved

Any center may submit cellular therapy data to the CIBMTR, and a single institution may have multiple centers. For more information, email [email protected].

2.1.4 CMS Coverage with Evidence Development (CED) Studies

Many patients with specific diseases and / or at certain ages are denied access to HCT therapy in the US due to lack of insurance coverage by the Centers for Medicare and

Medicaid Services (CMS).

Table 2.10 CMS CED Studies CMS CED studies allow CMS to provide coverage to patients enrolled on clinical studies that inform policy decisions. The CIBMTR is currently engaged in 5 CMS CED studies (Table 2.10). For additional information, visit the CIBMTR Medicare Clinical Trials webpage.

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Disease Patient Population Enrollment Dates

Myelodysplastic Syndrome (MDS)

(10-CMS-MDS)

NCT# 01166009

Elderly patients with MDS

132 centers

3,963 patients

2,530 patients ≥65 years old

1,205 patients 55-64 years old

228 patients <54 years old

Launched in 2010

Sickle Cell Disease

(BMT CTN 1503)

NCT# 02766465

Adolescents and young adults with severe sickle cell disease

38 centers

70 patients

Launched in October 2016

Myelofibrosis

(16-CMS-MF)

NCT# 02934477

Patients aged ≥55 years with primary myelofibrosis or postessential thrombocythemia / polycythemia vera

109 centers

112 patients

25 related (matched 6/6)

74 unrelated (matched 8/8)

13 haploidentical

Launched in December 2016

Multiple Myeloma

(17-CMS-MM)

NCT# 03127761

Elderly patients with Stage II or III multiple myeloma or primary plasma cell leukemia who are eligible to receive allogeneic HCT

80 centers

5 patients (550 planned)

Launched in July 2017

Sickle Cell Patients aged 15-50 69 centers Launched in Disease years with severe sickle 3

patients (200 planned) November 2017

(17-CMS-SCD) cell disease

NCT# 01166009


2.1.5 Patient-Reported Outcomes

The CIBMTR’s electronic patient-reported outcomes (ePRO) system, managed by staff in the Survey Research Group (Section 2.3.2), integrates three applications: A user-friendly interface in Qualtrics®; automated tracking and alerting functionality provided via the CIBMTR’s contact management system, which communicates directly with FormsNet; and computer adaptive testing that decreases respondent burden by only presenting patients with PROMIS (Patient Reported Outcome Measurement Information System) questions relevant to them.

The system is scalable, allowing additional measures, such as financial toxicity and employment, to be collected to study longterm survivorship issues identified by patients and caregivers. PRO data collected centrally through this system will place no additional burden on centers and, with the patient’s permission, can be returned to

centers using functionality of the Enhanced Data Back to Centers (eDBtC) platform (Section 3.2.3). The plan is to characterize the patient experience, overall and in specific patient cohorts defined by disease, ethnic, racial, region, age, and other demographics.

In 2018, the CIBMTR launched a pilot project to obtain PRO electronically using its new system. The CIBMTR approaches patients with the study and obtains consent electronically through the ePRO system. As of December 2018, the pilot project enrolled 28 patients from 2 centers. The primary objective is to compare quality of life in HCT recipients age 55-64 with recipients age 65 and older. Secondary objectives test the feasibility of ePRO collection, using the new CIBMTR ePRO system, in recipients age 55 and older. 2.1.6 International Initiatives

This year the CIBMTR continued to strengthen its international collaborations with clinical centers and international registries.

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European Society for Blood and Marrow Transplantation. The CIBMTR and EBMT collaborate to collect and exchange data from cellular therapy teams, share data for research studies, and define and revise common data elements and collection instruments. The combined resources of the registries have produced high quality studies with significant impact that are unlikely to have been possible in single center or single registry studies.

Until there is a better understanding of the impact of the new European Union General Data Protection Requirement (GDPR) legislation, EBMT data submission has proven to be challenging and data collected via AGNIS (A Growable Network Information System) is currently on hold. However, the CIBMTR remains committed to this collaboration and is working with its EBMT colleagues to determine a solution (Section 4.4.2).

Worldwide Network for Blood and Marrow Transplantation (WBMT). The WBMT collaborates with CIBMTR leaders in a wide range of areas but with a particular focus on harmonizing data collection forms and specific data elements to share transplant data worldwide. These data are used in reports of international transplant utilization and to support meaningful health services research. The CIBMTR and WBMT are utilizing these same processes to define standards for data collection for emerging indications of cellular therapy.

In April 2018, 3 CIBMTR Scientific Directors spoke at the WBMT Workshop and Scientific Symposium in Casablanca, Morocco. In

September 2018, 3 CIBMTR Scientific Directors spoke at the WBMT Workshop and Scientific Symposium in Beijing, China. World Marrow Donor Association (WMDA). WMDA has a committed partnership with the CIBMTR in a global aspect of processing

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standardization and data collection and sharing – all with the vision of improved outcomes of HCT.

Since WDMA’s inception, the CIBMTR has worked closely with the organization to improve global accessibility to donors for unrelated recipients in need of HCT, standardize global identification systems to match donors and recipients for data reporting, and conduct important research about donor outcomes.

Canadian BMT Group and Japan Society for HCT. The CIBMTR partners with the Canadian BMT Group and Japan Society for HCT to collect data from centers in Canada and Japan and return those data to the regional groups to create their own national outcomes registries. Using this approach, the regional groups leverage the CIBMTR’s data collection infrastructure and provide the CIBMTR with valuable international data.

Data Management. The CIBMTR offers training opportunities to physicians and data managers of international centers. Representatives are encouraged to visit the Milwaukee and Minneapolis campuses and to receive focused training on data use, study design, and statistical methods. The CIBMTR also conducts on-site trainings at international sites. In August 2018, CIBMTR staff members along with a group of Brazilian data managers provided training at the Brazilian Data Management Conference held in conjunction with the Brazilian Blood and Marrow Transplant Annual Meeting in Rio de Janeiro.

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2.2 IMMUNOBIOLOGY RESEARCH PROGRAMThe CIBMTR maintains a Research Repository of paired tissue samples from donors and recipients, both unrelated and related. Immunobiology Research Operation manages the Research Repository inventory and immunogenetic testing programs that add critical HLA and killer-cell immunoglobulin-like receptors (KIR) data for use in CIBMTR clinical outcomes studies.

The CIBMTR leverages the NMDP/Be The Match’s investment in the Unrelated Donor Research Repository with the NIH’s investment in the CIBMTR Research Database. Linking outcomes data to immunologic data available in the Research Repository supports studies that include genetic and immunobiologic data and clinical phenotype data.

The Related Donor Research Repository, supported by HRSA, is a unique opportunity to enhance immunobiologic research. Related donor and recipient samples are better matched than unrelated recipients for HLA, a measure of immunological compatibility, thus reducing the confounding effects of HLA disparity in clinical research.

The combination of the Unrelated and Related Donor Research Repositories facilitates an organized approach to studying transplant biology across the spectrum of allogeneic HCT. Program Activities

In 2018, 206 centers (146 transplant centers, 38 donor centers, and 22 cord blood banks) provided samples to the Research Repository. Immunobiology Research Operations enhanced the Research Repository inventory and Immunogenetic Database this year by

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completing high resolution HLA and KIR typing on 997 related and 3,195 unrelated HCT donor / cord and recipient pairs, bringing the total to >32,000 unrelated donor / cord and

Research Repository

2,625,472 aliquots

17,995 cell lines

70,992 samples from unrelated donors and 9,211 from related donors

63,678 samples from unrelated recipients and 9,579 from related recipients

12,153 samples from unrelated cord blood units

Samples from complete pairs:

41,102 from complete unrelated adult donor-recipient pairs

8,106 from complete related donorrecipient pairs

5,628 from unrelated cord-recipient pairs

recipient pairs that have been retrospectively high resolution typed for HLA-A, -B, -C, -DRB1 and -DQB1; >80% include -DPB1, and >18,000 include KIR.

Immunobiology Research Operations distributed 10,688 research samples in support of Working Committee studies this year. Publications

The Immunobiology Research Program supports investigators’ publications by providing research samples. 22 manuscripts published this year by Working Committee, BMT CTN, and Bioinformatics investigators

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Repository. Presentations

Immunobiology Research Program investigators presented 2 abstracts (1 oral and 1 poster) at national and international conferences in 2018. A complete list of CIBMTR presentations is

E.

Funding

Support for the Immunobiology Research Program is primarily provided by the Office of Naval Research grant # N00014-1-1-2045, NIH grant #

The Immunobiology Research Program offers limited research funds supporting immunobiology research studies. The grants are intended to subsidize lab tests, sample collection, or costs associated with the use of research samples. These grants are available to

How to Get Involved

All interested parties may attend the annual in-person meeting of the Immunobiology Working Committee at February. Additionally, the Immunobiology Working Committee encourages highly translational, hypothesis-

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Key Publications supported by the Immunobiology

Research Program this Year

Shaw BE, Logan BR, Spellman SR, et al. Development of an unrelated donor selection score predictive of survival after HCT: Donor age matters most. Biology of Blood and Marrow Transplantation. 2018 May 1; 24(5): 1049-1056. Epub 2018 Feb 14. PMC5953795.

Zhu Q, Yan L, Liu Q, et al. Exome chip analyses identify genes affecting mortality after HLA-matched unrelateddonor BMT. Blood. 2018 May 31; 131(22): 2490-2499. Epub 2018 Apr 2. PMC5981168.

Gadalla SM, Aubert G, Wang T et al. Donor telomere length and causes of death after unrelated HCT in patients with marrow failure. Blood. 2018 May 31; 131(21): 2393-2398. Epub 2018 Apr 9. PMC5969378.

Petersdorf EW, Stevenson P, Malkki M et al. Patient HLA germline variation and transplant survivorship. Journal of Clinical Oncology. 2018 Aug 20; 36(24): 2524-2531. Epub 2018 Jun 14. PMC6097831.


driven proposals through the Working Committee Study Proposal Review Process (Figure 2.9).

2.3 CLINICAL TRIALS SUPPORT PROGRAMThe CIBMTR manages a wide array of studies, including multi-center trials, surveys, and correlative studies. Access to the CIBMTR Research Database and use of data from observational studies are important resources to support decisions regarding design of prospective clinical trials.

CIBMTR Coordinating Center Support of Clinical Trials

Study Planning. Oversee study development, including patient population identification, site selection, training, and financial administration.

Data Collection. Collect new data and collaborate to share existing data across systems and centers.

Site Management. Oversee site startup, enrollment, and protocol compliance.

Study Monitoring. Oversee onsite, centralized, and remote monitoring to ensure data accuracy and mitigate risks.

Statistical Consultation. Provide expert design and review of protocols.

Real-Time Accrual Assessment. Review characteristics of enrolled and non-enrolled patients to address potential

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accrual barriers.

Trial Interpretation. Evaluate results of clinical trials, including through the provision of matched controls.

Long-Term Follow-Up Data. Capture follow-up data for long-term or secondary analyses, resulting in considerable cost-savings.

2.3.1 Blood and Marrow Transplant Clinical Trials Network

The BMT CTN, sponsored by NHLBI and NCI, is the US network charged with developing and conducting multicenter Phase II and III clinical trials focused on cellular therapy. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation, a contract research organization based in Rockville, MD. Program Activities

The BMT CTN has launched 46 trials and completed accrual for 40 of these trials (6 this year). The Network has accrued >10,600 patients to its trials from >100 centers, including >550 this year. The Network has established a Research Sample Repository that currently includes >408,000 biospecimens. Additionally, the BMT CTN has conducted 46 ancillary and correlative studies, with another 40 in progress.

More detail regarding Network activities and protocols is provided in the annual Progress Report on the BMT CTN website. A list of Network trials open for enrollment is provided in Appendix G1.

Publications

In 2018, BMT CTN study investigators published 13 manuscripts, all of which were peer-reviewed journal articles. These bring the total number of Network publications to 95,

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including 25 primary results papers. A complete list of 2018 BMT CTN publications is provided in Appendix D2.

Presentations

BMT CTN study investigators presented 3 abstracts (1 oral and 2 poster) at national and international conferences in 2018. These bring the total number of Network presentations to

94. A complete list of 2018 CIBMTR presentations is provided in Appendix E. Funding

Support for the BMT CTN Data and Coordinating Center is provided by the NIH grant # U24HL138660 from the NHLBI and NCI. How to Get Involved

The Network is committed to widespread participation in its trials. If you would like to serve as an Affiliate Center, visit the BMT CTN website for more information. Additionally, you may act as a Center Principal Investigator or champion a trial to increase patient accrual at your Center, serve on a Protocol Team or an Endpoint Review Committee, or act as a Medical Monitor. You may also propose an

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Key BMT CTN Publications this Year

Carpenter P, Logan BR, Lee, S et al. A Phase II/III randomized, multicenter trial of prednisone / sirolimus vs. prednisone / sirolimus / calcineurin inhibitor for treatment of chronic GVHD: BMT CTN 0801. Haematologica. 2018 Nov 1; 103(11): 1915-1924. Epub 2018 Jun 28. PMC6278959.

Allen C, Marsh R, Dawson P et al. Reduced intensity conditioning for HCT for HLH and primary immune deficiencies. Blood. 2018 Sep 27; 132(13): 1438-1451. Epub 2018 Jul 11. PMC6161764.

Rashidi A, Shanley R, Yohe SL et al. Recipient SNP in Paneth cell antimicrobial peptide genes and acute GVHD: Analysis of BMT CTN-0201 and -0901 samples. British Journal of Haematology. 2018 Sep 1; 182(6):887894. Epub 2018 Jul 13. PMC6128755.

Rashidi A, Holtan, S, Bhatt, A et al. Antibiotic practice patterns in HCT: A survey of BMT CTN centers. American Journal of Hematology. 2018 Nov 1; 93(11): E348-E350. Epub 2018 Jul 30. PMC6196101.


ancillary study, which uses data, biospecimens, and / or analyses outside the specific objectives of a primary BMT CTN study.

2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation

The RCI BMT provides cellular therapy researchers with infrastructure and expertise in clinical trial conduct and analysis. The program not only helps investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting but also supports Phase II/III trials and large survey and cohort studies. Program Activities

The RCI BMT has launched 18 studies, including 1 this year. In 2018, the RCI BMT accrued >2,500 patients, bringing the total number of accrued patients to approximately 36,000, of which >21,000 were enrolled in a cohort study examining long-term outcomes of unrelated donors.

The RCI BMT manages 2 FDA investigational new drug (IND) protocols for NMDP/Be The Match. PBSC Procurement accrued >1,400 subjects this year, and Cord Blood Access accrued >400. These protocols allow US centers to access peripheral blood and unlicensed cord blood for transplantation.

The RCI BMT also supported 6 active studies and participated in the development of 4 upcoming studies. A complete list of RCI BMT studies is provided in Appendix G2.

Survey Research Group

The Survey Research Group is a team within the RCI BMT created to assist researchers in developing and conducting research involving questionnaires, direct subject interviews, and patient reported outcomes. The group is

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responsible for collecting high quality, scientifically valid data from donors, patients, and their families. In 2018, the Survey Research Group launched a new ePRO system (Section 2.1.5) to support clinical trials, long-term follow-up, and other research studies with patients and donors. The groups also supported 4 active studies and participated in the development of 5 upcoming studies.

Publications

In 2018, RCI BMT study investigators published

2 peer-reviewed manuscripts in Biology of Blood and Marrow Transplantation. A list of RCI BMT publications is provided in Appendix D3.

RCI BMT Publications this Year

Nemecek ER, Hilger RA, Adams A, et al. Treosulfan, fludarabine and low-dose TBI

for children and young adults with AML or MDS undergoing allogeneic HCT: A prospective Phase II trial of the PBMTC. Biology of Blood and Marrow Transplantation. 2018 Aug 1; 24(8):1651-1656. Epub 2018 May 9. PMC6108922.

Jacobsohn DA, Loken MR, Fei M. Outcomes of measurable residual disease in pediatric AML pre- and postHCT: Validation of difference from normal flow cytometry with chimerism studies and Wilms tumor 1 gene expression. Biology of Blood and Marrow Transplantation. 2018 Oct 1; 24(10): 2040-2046. Epub 2018 Jun 19. PMC6239928.

Funding

Support for RCI BMT studies is provided by NMDP/Be The Match, corporate and private sponsors of specific studies, and via grant mechanisms.

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The RCI BMT team can work with study investigators to seek funding from a variety of sources, including government agencies, foundations, pharmaceutical companies, and private corporations.

How to Get Involved

Study investigators may solicit clinical trials services from the RCI BMT, including assistance with funding proposals; protocol development and approvals; management of study conduct; data auditing, management, and analysis; and financial administration. Study investigators may also contract for specific services as needed, such as support with surveys, site selection and management, sample management, and more. For additional information, visit the RCI BMT webpage.

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2.4 HEALTH SERVICES RESEARCH PROGRAMHealth services research is the multidisciplinary field of scientific investigation that studies how social factors, financial systems, organizational structures and processes, technology, and behavior affect treatment outcomes, quality, and cost. Program Activities

The Health Services Research Program currently has 8 studies in progress. In 2017, the program completed analysis for 7 research studies.

Select Health Services Research Studies in Progress

Reimbursement analysis of HCT costs in older patients

Costs of HCT by transplant setting

Stepped-care approach for HCT survivors

Patient-reported outcomes

Patient perspective regarding which clinical trial outcomes to share

Health Care Disparities. In collaboration with the University of Virginia, the Health Services Research Program identified the unmet need for allogeneic HCT of patients in the state of Virginia with correlation by regional socioeconomic status. The program also launched an analysis of Medicaid coverage of HCT for sickle cell disease in collaboration with the NMDP/Be The Match Public and Payer Policy Program.

Health Economics. In collaboration with NMDP/Be The Match’s Public and Payer Policy Department, the Health Services Research Program conducted 3 studies to evaluate

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reimbursement of HCT-related health services. Using Optum Clinformatics data, investigators compared costs of HCT versus chemotherapy alone for adults with de novo acute myeloid leukemia (AML). Medicare data was linked to the CIBMTR Research Database to analyze reimbursement and utilization for older patients diagnosed with AML, and the program partnered with investigators from Kansas University to explore cost of readmissions following HCT. Program analysts demonstrated the feasibility of linking the CIBMTR Research Database to the Vizient database for future research in health economics.

Survivorship and Quality of Life. In 2018, the Health Services Research Program reported the results of their efforts to develop a patientcentered outcomes HCT research agenda. Working groups comprised of patients, family members, and health care providers prioritized comparative

effectiveness research questions in 6 topic areas. Central to this project was the engagement of patients and caregivers in all aspects of planning, implementation, and dissemination.

Patient-Centered Outcomes Research Topic Areas

Physical health and fatigue

Emotional, cognitive, and social health

Financial burden

Models of care delivery - survivorship and late effects

Patient, caregiver, and family education and support

Sexual health and relationships

The Program also reported the results of a

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multi-year, prospective randomized study that utilized the CIBMTR Research Database to prepare individualized survivorship care plans for HCT survivors. This work was a collaborative effort with investigators from Cleveland Clinic and the Fred Hutchinson Cancer Center. The program continues a collaboration with these same investigators in the INSPIRE project, which provides stepped care self-management program for survivors. The Health Services Research Program is collaborating with the RCI BMT (Section 2.3.2) and multiple transplant centers to prospectively collect quality of life PROs from patients who have undergone HCT for MDS.

Treatment Decision-Making Support. The Health Services Research Program reported results of the impact of BMT CTN 0201 research findings on transplant physicians’ selection of graft source. An ongoing project with ASH targeting the multidisciplinary care team that cares for HCT patients broadened

its scope to include both MDS and AML; a special education session was held at the 2018 ASH Annual Meeting. The Program completed a multi-year project that identified knowledge gaps among community physicians who care for patients with AML and developed a series of 3 webinars that addressed these gaps, including diagnosis and risk stratification and timing of referral for HCT consultation. Publications

In 2018, Health Services Research investigators published 6 peer-reviewed manuscripts in medical journals. A list of program publications is provided in Appendix D4. Presentations

In 2018, program investigators presented 6 abstracts (3 oral and 3 poster) at national and international conferences. A complete list of CIBMTR presentations is provided in Appendix E.

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Key Health Services Research Publications this Year

Burns LJ, Abetti B, Arnold SD, et al. Engaging patients in setting a patientcentered outcomes research agenda in HCT. Biology of Blood and Marrow Transplantation. 2018 Jun 1; 24(6): 11111118. Epub 2018 Feb 3. PMC5993588.

El-Jawahri A, LeBlanc TW, Burns LJ, et al. What do transplant physicians think about palliative care? A national survey study. Cancer. 2018 Dec 1; 123(23): 45564566. Epub 2018 Oct 5. PMC6289734.

Denzen EM, Preussler JM, Murphy EA, et al. Tailoring a survivorship care plan: Patient and provider preferences for recipients of HCT. Biology of Blood and Marrow Transplantation. Epub Oct 10.

Funding

Health Services Research Program studies are funded via a variety of mechanisms. Engaging patients in developing a patient-centered HCT research agenda was supported by the Patient-Centered Outcomes Research Institute (PCORI) engagement award # EAIN-2956. Individualized care plans for HCT survivors was supported by the PCORI award # CD-12-114062. A payer-partnered approach to community-based referral for HCT was supported by the grant # 11762021 from the National Comprehensive Care Network / Pfizer. INSPIRE is funded by the NIH grant # R01CA215134-01 from the NCI. Additional support for the Health Services Research Program is provided by NMDP/Be The Match.

How to Get Involved

For more information about the Health Services Research Program, contact Linda Burns, MD, Senior Scientific Director, at [email protected].

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2.5 BIOINFORMATICS RESEARCH PROGRAMThe Bioinformatics Research Program provides expertise in, and conducts research on, translational and operational bioinformatics. Program Activities

Current Bioinformatics Research Goals

Develop pipelines to analyze Next Generation Sequencing typing data, including full-gene HLA, KIR, and genomewide sequencing, to refine our understanding of genetic matching

Investigate the role of genetic ancestry in transplantation, including the best way to match multiracial individuals

Develop data standards and tools for making immunogenetic data portable for research and clinical use

Investigate HLA data from other countries to better understand global frequencies and improve matching

Develop methods for HLA association studies

Publications

In 2018, Bioinformatics study investigators published 11 peer-reviewed manuscripts in scientific journals. A complete list of program publications is provided in Appendix D5. Presentations

Bioinformatics study investigators presented 15 abstracts (10 oral and 5 poster) at national and international conferences in 2018. A complete list of CIBMTR presentations is provided in Appendix E.

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Key Bioinformatics Publications this Year

Roelen D, de Vaal Y, Vierra-Green C et al. HLA mismatches that are identical for the antigen recognition domain are less immunogenic. Bone Marrow Transplantation. 2018 Jun 1; 53(6): 729740. Epub 2018 Feb 6.

Halagan M, Oliveira D, Maiers M et al. The distribution of HLA haplotypes in the ethnic groups that make up the Brazilian Bone Marrow Volunteer Donor Registry (REDOME). Immunogenetics. 2018 Aug 1; 70(8): 511-522. Epub 2018

Apr 26.

Sivasankaran A, Williams E, Switzer GE et al. Machine learning approach to predicting stem-cell donor availability. Biology of Blood and Marrow

Transplantation. 2018 Dec 1; 24(12): 2425-2432. Epub 2018 Jul 31.

Funding

Support for the Bioinformatics Research Program is primarily provided by the grant # N00014-17-1-2045 from the Office of Naval Research. Integrated Exchange and Storage of Current and Future-Generation Immunogenomic Data is supported by the R01 grant # AI128775 from NIAID. How to Get Involved

For more information about the Bioinformatics Research Program, contact Martin Maiers,

Vice President of Biomedical Informatics, at [email protected] or 612.627.5892.

The CIBMTR has enjoyed a positive, collaborative association with the Division of

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2.6 STATISTICAL METHODOLOGY RESEARCH PROGRAM


Biostatistics in the MCW Institute for Health and Equity since 1980, an association that is a distinctive asset and crucial to the success of CIBMTR research. Biostatisticians ensure the statistical integrity of CIBMTR scientific activities, contribute to results in articles on cellular therapy-related statistical issues for clinical audiences, and support Working Committee study investigators in developing scientific study protocols using CIBMTR data. CIBMTR biostatisticians have pioneered novel methodologic approaches to analyzing cellular therapy data.

Program Activities

Transplantation is a complex process with multiple competing risks and dramatic changes in the risks of specific events over time. The CIBMTR has developed and evaluated the statistical models used in cellular therapy research and helped guide the research community in appropriate application and interpretation of these

sophisticated models.

Statistical Methodology Research Goals

Develop new statistical models

Compare new statistical models with existing solutions using the CIBMTR Research Database

Publications

In 2018, PhD-level biostatisticians in the

CIBMTR published 3 peer-reviewed statistical methodology manuscripts. A list of program publications is provided in Appendix D6.

Key Statistical Methodology Publications this Year

Ahn KW, Kim S. Variable selection with group structure in competing risks quantile regression. Statistics in

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Medicine. 2018 Apr 30; 37(9):1577-1586. Epub 2018 Feb 21. PMC5889760.

Wang Y, Logan BR. Testing for center effects on survival and competing risks outcomes using pseudo-value regression. Lifetime Data Analysis. Epub 2018 Jul 5. PMC6320737.

Presentations

Statistical Methodology Research Program investigators presented 4 oral abstracts at national and international conferences in 2018. A complete list of CIBMTR presentations is provided in Appendix E. Funding

Support for the Statistical Methodology Research Program is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID and the HRSA contract # HHSH250201700006C.

How to Get Involved

During the TCT Meetings in February, PhD-level biostatisticians plan and present educational sessions related to statistical design and analysis, and they provide 1:1 statistical consultation to researchers writing proposals or developing protocols for CIBMTR studies. Any interested individual may participate in these sessions. Additionally, the MCW Division of Biostatistics presents a lecture series and a seminar series throughout the year in Milwaukee. 2.7 CORPORATE PROGRAMThe CIBMTR provides support to industry, a critical partner in achieving our mission, through the CIBMTR Corporate Program’s Corporate Memberships as well as Studies and Projects. Through these partnership agreements, industry can leverage the expertise of CIBMTR statistical support, center network, database, information technology (IT) and data management systems, and

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scientific leadership. Requests are considered based on their alignment with CIBMTR’s mission and feasibility.

Corporate Membership. The CIBMTR Corporate Membership program provides a variety of resource materials to corporations needing access to the most current and comprehensive data on cellular therapy. These materials are useful for Marketing Managers, Medical Directors, Research Directors, Product Managers, Case Managers, and Transplant

Coordinators. The CIBMTR offers 5 levels of Corporate Membership, each described on the CIBMTR Corporate Membership Program webpage.

Corporate Membership Benefits

CIBMTR Report on Survival Statistics for BMT

Center Volumes Dataset

Worldwide CIBMTR Directory of BMT Physicians

Reduced registration rates at CIBMTR meetings and educational forums, including the TCT Meetings

Access to CIBMTR data and resources Corporate Studies and Projects. Corporate Studies and Projects may be one-time requests or long-term projects and studies. Services include: Descriptive reports and analysis of data beyond corporate membership access, raw patient-level data, protocol development, registry development and management, retrospective and prospective studies, consultation services, supplemental data collection, and large-scale, collective impact projects. Corporate requests may also ask for a one-time descriptive report comparing HCT patient characteristics from a clinical trial cohort with a matched historical control patient population from

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the CIBMTR Research Database.

Program Activities

The CIBMTR engaged in 18 studies with corporate partners in 2018. Currently 21 organizations participate in the CIBMTR Corporate Membership program.

How to Get Involved

If you would like to learn more about the CIBMTR Corporate Program, visit the CIBMTR Corporate Membership Program webpage or contact Sherry Fisher, Director of Business Development, at [email protected] or 414.805.0687. If you are a Corporate Member requesting analyses, please complete the Corporate Member Information Request Form on the CIBMTR website.

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE

3.0 HOW WE SHARE KNOWLEDGE

The CIBMTR is committed to sharing the data we collect as well as the information and knowledge produced from our data and our extensive collaborations with investigators in the cellular therapy field.

The CIBMTR shares its knowledge in different ways. To determine the best way to access specific types of CIBMTR knowledge, review Figure 3.1 and Tables 3.2-3.5.

Figure 3.1 How to Access CIBMTR Knowledge

Details provided in Tables 3.2-3.5.

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Table 3.2 How to Access Information Table 3.3 How to Access Data Online at Online at cibmtr.org* cibmtr.org*

Information Data

GENERAL INFORMATION In addition to reviewing this report, access the Facts and Figures report on the Administrative and Progress Reports webpage. Read editions of the quarterly newsletter on the Newsletters webpage, and email [email protected] to be added to the electronic distribution list.

ACTIVITIES Review Section 2 or visit the What We Do webpage. Visit the Studies webpage, SCTOD webpage, or Corporate Support webpage to learn more about CIBMTR research programs, the SCTOD, or the Corporate Program, respectively.

MEETINGS Visit the Meetings webpage for information about annual TCT Meetings and other major events, such as Cellular Therapy and Center Outcomes Forums.

TRAINING Review Section 3.4 or visit the Training and Reference webpage to access the Data Management Guide, Forms Instruction Manual, FormsNet and AGNIS trainings, and online courses.

PUBLICATIONS Review the CIBMTR’s >1,300 publications on the Publication List webpage. For summaries of selected CIBMTR publications written specifically for patients and the lay public, visit the Study Summaries for Patients webpage.

OTHER Email [email protected]

TYPES OF DATA Review the baseline and follow-up data available for recipients and donors on the Types of Data Available for Research or Request webpage. Assess data fields available in the Research Database by reviewing the Data Collec tion Forms for Investigators webpage.

STANDARD REPORTS Access the Summary Slides, BMT Survival Statistics Report, Center Transplant Activity Report, Patient Transplant Outcomes Reports, and Center-Specific Survival Reports on the Slides and Reports webpage.

RESEARCH STUDIES Propose a study as explained on the How to Propose a Study webpage, or participate in one of the existing studies listed on the Working Committee Study Lists webpage.

RESEARCH DATASETS To request a dataset, propose a study as explained on the How to Propose a Study webpage. To access final study analysis datasets prepared for published studies, visit the Publication List webpage.

CUSTOM ANALYSES Complete the Custom Information Request Form or, for Corporate Members, the Corporate Member Information Request Form.


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Table 3.4 How to Access Tools Online at cibmtr.org*

Table 3.5 How to Access Biospecimens Online at cibmtr.org*

*If you are unable to access items using the electronic links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).

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More Tools

VOD RISK CALCULATOR Access this tool on the VOD Risk Calculator webpage to identify patients at high risk for veno-occlusive disease (VOD).

Tools

Biospecimens

SAMPLES TYPES AND INVENTORY Review the >2 million samples available in the Research Repository via the Sample Types and Inventory Summary webpage.

REQUESTING SAMPLES For studies that include recipient clinical outcome data, propose a study as explained on the How to Propose a Study webpage. For studies that do not include clinical outcome data, review the How to Request Samples from the Research Sample Repository webpage.


CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Table 3.6 Standard Reports Published by the CIBMTR

Report Title Description Accessibility* CIBMTR Annual Report

Information on the CIBMTR's goals and achievements as well as operational details on how the CIBMTR is funded, supported, promoted, and maintained

Published on the CIBMTR Administrative and Progress Reports webpage Released: February Format: PDF

CIBMTR Summary Slides

Charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT; developed in conjunction with the TCT Meetings

Published on the CIBMTR Summary Slides webpage Released: February Format: PPT

CIBMTR Facts and Figures High level summary of CIBMTR fiscal year accomplishments and high impact publications

Published on the CIBMTR Administrative and Progress Reports webpage Released: September Format: PDF

US Centers Annual Transplant Activity Report

Dataset containing center-specific pretransplant patient-, disease-, and transplant-related characteristics data for nearly all allogeneic and a majority of autologous HCTs performed in the US annually since 2008

Published on the HRSA CWBYCTP website Released: September Format: PDF

CIBMTR Report of Survival Statistics for BMT

Highly detailed report on survival statistics that describes use and outcome of autologous and allogeneic HCT in the >500 centers that have participated in the CIBMTR

Via Corporate Membership Program (Section 2.7) or by request from physicians for making treatment decisions or clinical investigators planning clinical studies to [email protected] Released: November Format: Word

US Patient CenterSpecific Survival Report

Comparison of observed to expected one-year survival rates among centers in the HRSA CWBYCTP network; evaluates outcomes for transplants using both related and unrelated donors

Published on the Be The Match Transplant Center Directory webpage; available as a Word document upon request to [email protected] Released: December Format: Web

Report Title Description Accessibility*

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE US Patient Transplant Outcomes

Disseminated in 3 different reports: • US Patient Survival Report: 100day, 1-year,

and 3-year survival rate estimates for US HCT recipients by disease and donor type

• US Transplant Data by Center Report: Number of bone marrow and cord blood transplants performed at a specific center

• US Transplant Data by Disease Report: Number of bone marrow and cord blood transplants for a specific disease

Published on the HRSA CWBYCTP website Released: December Format: Web

US Allogeneic Transplant Activity Report

Report containing patient, disease, donor HLA match, donor age, and gender match information for allogeneic transplant activity in the US since 2010

Via Corporate Membership Program (Section 2.7) Released: January, April, July and October Format: PDF

CIBMTR Newsletter Articles regarding Working Committees, the SCTOD, data management and collection, and noteworthy events in the cellular therapy community

Published on the CIBMTR Newsletters webpage and distributed via email; contact [email protected] to be added to the distribution list Released: February, May, August and November Format: Web

Study Summaries for Summaries of CIBMTR research Published on the CIBMTR Study Patients publications written for patients and Summaries for Patients webpage

others in the lay public Released: Ongoing Format: PDF

*If you are unable to access items using the electronic links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org).

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.1 INFORMATION REQUEST SERVICEThe CIBMTR Information Request Service provides timely access to cellular therapy data to patients, physicians, hospitals, pharmaceutical companies, insurance companies, and others involved in healthcare. Requests range from simple queries of patient, disease, and therapy frequencies to those with greater complexity involving specific data combinations and / or statistical analysis of outcomes.

Potential Reasons for Information Requests

Self-education and decision making

Patient counseling or clinical decision making

Presentation support

Center assessments

Clinical trial planning

Market assessments

Coordinating Center staff members fulfill requests related to clinical decision making within three days and most other requests within three weeks. If a request will take more than an estimated four weeks to fulfill, a Coordinating Center staff member will contact the requestor to discuss an appropriate timeline.

Accomplishments

In 2018, the CIBMTR fulfilled 433 requests for information and data (Table 3.7). Table 3.7 Data Requests Addressed by the CIBMTR in 2018

Requestor Number of Requests

Physician / Researcher 281

Commercial Organizations 57

Physician for Patient Care 39

Student 19

Market Research Firm 15

Patient or Relative 12

Federal Government Agency 5

Patient Advocacy Group 2

Cord Blood Bank 1

Law Firm 1

Insurance Company 1

TOTAL 433

How to Access

For more information about requesting data from the Research Database, visit the CIBMTR How to Request Data webpage. If you would like a one-time, custom analysis, complete the Custom Information Request Form on that webpage. If you have questions about requesting CIBMTR data, please contact [email protected].

3.2 INTERNET PRESENCEThe CIBMTR Internet presence provides the scientific community and the public with access to cellular therapy information. Current websites include general information about cellular therapy and CIBMTR activities; training and support; a shared communications and collaborative environment for member centers; and secure web access to CIBMTR data for committees, centers, scientific investigators, and CIBMTR staff members.

3.2.1 CIBMTR Public Website

The CIBMTR public website (cibmtr.org) is unrestricted and provides information about the CIBMTR and its research. It supports the

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Working Committees and BMT CTN with information regarding proposal submission, access to a listing and summaries of all studies in process, and access to a summary of all CIBMTR publications. The website facilitates data and information requests, and it provides access to all current and past data collections forms, training manuals, and videos as well as other materials for both investigators and data professionals. The website information is, in part, supported by DISCO (Data and Information for Statistical Center Operations), an application which maintains data on >900 studies, >1,300 publications, and >2,700 authors and their institutions at time of publication. In 2018, the CIBMTR public website had 620,970 unique page views.

About CIBMTR

Administrative and Progress Reports

(1,534 unique page views in 2018) Provides access to the CIBMTR’s Annual Progress Report, Manual of Operations, and Facts and Figures document.

Studies

Working Committee Studies Lists

(4,644 unique page views in 2018) A summary of the planned, in-progress, and recently published clinical outcomes studies for each Working Committee. Meetings

Meeting Materials

(21,595 unique page views in 2018) Provides access to agendas, handouts, and educational materials from specific meetings at the TCT Meetings, such as Working Committee Meetings and Clinical Research Professionals / Data Management Conferences, as well as other major events, such as Cellular Therapy and Center Outcomes Forums.

Reference Center

Summary Slides

(11,703 unique page views in 2018) Includes charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT.

Web-based US Transplant Reports

(19,807 unique page views in 2018) Directs users to the Be The Match US Center Listing Report and customizable reports of patient survival and transplant available through the HRSA CWBYCTP website.

Publication List

(5,993 unique page views in 2018) Searchable descriptive list of >1,300 publications resulting from the use of CIBMTR data and statistical resources. Newsletters

(4,891 unique page views in 2018) Published 4 times per year. Articles feature updates on Working Committees, the SCTOD, data management and collection, and noteworthy events in the cellular therapy community.

Patient Resources

(5,913 unique page views in 2018) Includes lay summaries of CIBMTR research articles as well as post-transplant care recommendations for adult and pediatric autologous and allogeneic HCT recipients to help patients and clinicians understand and plan for the specialized care of transplant recipients. The CIBMTR published 23 study summaries for patients in 2018.

Statistical Resources

(8,810 unique page views in 2018) Provides access to resources offered through the unique partnership between the CIBMTR and MCW Division of Biostatistics, including

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE biostatistical publications, a series of statistical lectures targeted at basic and clinical investigators, and research tools, such as the DRI Assignment Tool and VOD Risk Calculator.

Data Management

Data Management Manual

(219,983 unique page views in 2018) A comprehensive reference document for completing CIBMTR data collection forms. The manual also details reporting requirements, describes protocols and the consent process, and includes downloadable report forms.

Data Back to Centers Applications

(703 unique page views in 2018) Links to the DBtC and eDBtC applications that provide CIBMTR member centers with selfservice access to their most commonly used CRF- and TED-level data, including descriptive statistics and outcomes. Data Collection Forms

(42,964 unique page views in 2018) Provides access to current and retired versions of forms used by the CIBMTR to collect standard data elements for all cellular therapy recipients.

Training and Reference

(68,327 unique page views in 2018) Provides access to a wide variety of CIBMTR data management training and reference materials.

3.2.2 CIBMTR Collaborative Site

The CIBMTR Collaborative site (collaborate.cibmtr.org) uses the SharePoint Enterprise Collaboration platform to promote cooperative work among CIBMTR staff members and provides a communication platform for specific studies and initiatives. This site is secured by username and password, and user-specific security credentials are assigned centrally.

The CIBMTR uses the site for storing and sharing protocol and consent documents, donor / recipient tracking tools, confidential committee information, data, manuscript drafts, and other relevant information. Several Working Committees use the Collaborate site on a regular basis, and all Working Committees are welcome to use the site for sharing information.

3.2.3 CIBMTR Portal Site

The CIBMTR Portal site (portal.cibmtr.org) delivers applications and data to CIBMTR centers and other partners. In 2018, external visitors viewed 30,701 Portal pages. 7 applications are currently hosted on this site:

Data Back to Centers

DBtC - Data Back to Centers The DBtC application provides authorized users the ability to download CIBMTR TEDlevel data variables for their centers. The data have been validated and processed in the CIBMTR Research Database and are reviewed and refreshed quarterly. Legacy International Bone Marrow Transplant Registry (IBMTR) data from as far back as 1964 and some legacy NMDP/Be The Match data from as far back as 1987 are available. In 2018, authorized users from 143 different centers viewed 2,506 DBtC pages.

eDBtC - Enhanced Data Back to Centers The CIBMTR deployed eDBtC in Qlikview in 2016 to provide centers with self-service access to their most commonly used CRF- and TED-level HCT data, including descriptive statistics and outcomes. eDBtC enables centers to view and filter outcomes, including a fiveyear overall survival curve as well as acute graft-versus-host disease (GVHD), chronic GVHD, and other outcomes, for the center's population.

An ad-hoc query tab allows users to create and implement their own custom query of these

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE data. eDBtC data are extracted from the CIBMTR Research Database and are validated, reviewed, and refreshed monthly. Users are also able to export filtered data in Excel file formats.

This year, the CIBMTR launched work to extend eDBtC beyond HCT and provide centers with access to data collected from their patients who received cellular therapy infusions. The CIBMTR plans to release this expanded scope and functionality next year. In 2018, 261 unique users from 189 different centers accessed 3,455 eDBtC sessions, and 159 users from 137 centers downloaded data 906 times. Data for RFI

The CIBMTR launched the Data for RFI (Request for Information) application in 2017 to help centers access and use the outcome data they share with the CIBMTR. Data for RFI provides centers with the ability to access, view, reconcile, and format data submitted to the CIBMTR for use in fulfilling submissions to third party payers and other organizations. Data for RFI leverages the same data available in eDBtC but translates these to the standard RFI format developed by the American Society of Blood and Marrow Transplantation (ASBMT). This application also incorporates the standard ASBMT RFI rules for determining survival and for differentiating between adult and pediatric populations. Centers can export data into the standard format developed by ASBMT and then supplement with additional data not collected by CIBMTR. In 2018, 142 unique users from 121 different centers accessed 687 Data for RFI sessions.

Center Performance Analytics

The CIBMTR deployed Center Performance Analytics in Qlikview in 2016 to support center performance and quality initiatives by allowing

authorized users to compare their center’s data to aggregated center data. Predefined filters for this comparison include geographic region, historical performance, volume of transplants as a proxy for size, and patient population served. Centers can also view their center's own one-year survival rate, based on a rolling three-year period of data included in the Transplant Center Specific Survival dataset. Like eDBtC, users can create and implement their own customized, ad hoc query and export a download of the source dataset for their center. In 2018, 97 unique users from 94 different centers accessed 377 Center Performance Analytics sessions.

Patient One-Year Survival Calculator for Allogeneic Transplants

The Patient One-Year Survival Calculator for Allogeneic Transplants provides authorized users with a tool to predict one-year survival for individual allogeneic HCT recipients. The calculator data are updated annually to reflect new information contained in the center outcomes analysis. In 2018, authorized users accessed the survival calculator 3,779 times.

Center Volumes Portal

The Center Volumes Portal allows centers to preview; correct, if necessary; and approve center volume data published annually on the HRSA CWBYCTP website. Centers may display and download the previous five years of volume data (2013-2017). In 2018, authorized users accessed the Center Volumes Portal 4,363 times.

Cord Blood Report Portal

The Cord Blood Report Portal provides secure, self-service access to monthly predefined Cord Blood Reports for authorized cord bank users. These reports, updated monthly, include data relevant to the quality and safety of distributed cord blood units to both domestic and international cord blood banks. Between its

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE release in September 2018 and December 2018, 31 unique users from 27 different centers accessed 223 Cord Blood sessions. 3.2.4 Be The Match Public Website

The Be The Match public website (bethematch.org) is designed for patients and families, donors, and supporters. It incorporates detailed information about transplantation and donation written for the public. The website provides scientific information in lay terms for donors related to the donation process and for patients related to specific diseases, various treatment options, the process of transplantation, and life after transplant. It also addresses concerns related to specific populations, including children and caregivers. The CIBMTR collaborates with NMDP/Be The Match to provide content for several areas of this website, including data for the US Center Listing Report. US Center Listing Report

Transplant Center Directory Provides transplant center specific information about facilities, personnel, diseases treated, cost, and transplant experience, including the number of transplants performed and survival rates by age, disease type, and disease stage.

3.2.5 Be The Match Clinical Website

The Be The Match Clinical website (bethematchclinical.org) is designed for clinicians, network participants, payors, and bioinformatics professionals. For clinicians, the website provides access to evidence-based tools, clinical guidelines, outcomes data, and education courses. The website also provides information specific to types of network participants: Transplant centers, donor centers, apheresis and collection centers, and cord blood banks. For payors, the website offers information to help individuals understand BMT, determine coverage, and answer employer and patient questions. Related to

bioinformatics, the website provides resources for immunogenetic-focused research and operational bioinformatics as well as frequently used HLA tools. 3.2.6 HRSA CWBYCTP Website

The HRSA CWBYCTP website (bloodcell.transplant.hrsa.gov) provides information for the public, physicians, and other constituents. It incorporates transplant resources, donor information, and cord blood information as well as research, data, and outcomes. CIBMTR data and research findings are incorporated in numerous ways, including through provision of datasets and CIBMTRcreated reports:

Transplant Outcomes and Data

US Patient Survival Report Provides disease-specific post-HCT survival estimates by the length of time after transplant: 100 days, 1 year, and 3 years. Survival estimates are also available by patient age, patient gender, patient race, or cell source.

Transplant Data by US Center Report Displays the number of adult donor and cord blood transplants performed at a specific center.

Transplant Data by Disease Report Displays the number of adult donor and cord blood transplants reported for a specific disease. Totals are also available by patient age, patient gender, patient race, cell source, and the year the transplant was performed.

Transplant Activity Report Displays the number of transplants performed at US centers, including autologous as well as related and unrelated allogeneic. Numbers are also available by patient age, patient gender, patient race, cell source, disease, center location by state, and year in which the transplant was performed.

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.2.7 Other Applications and Data Exchange Standards

The CIBMTR has multiple methods for sharing data. Those hosted on the CIBMTR Portal site (DBtC, eDBtC, Data for RFI, Center Performance Analytics, One-Year Survival Calculator, Center Volumes Portal, and Cord Blood Report Portal) were described in Section 3.2.3. Other methods for sharing data are: AGNIS

AGNIS allows participating centers to electronically collect and share data with the CIBMTR and others who link to AGNIS. Data are entered once and then distributed and synchronized among databases. In 2018, 19,573 forms for 9,772 patients were submitted through AGNIS by 27 US centers and by EBMT for 48 of their affiliated centers.

BRIDG

The BRIDG (Biomedical Research Integrated Domain Group) Model is an information model, representing a shared view of the concepts of basic, pre-clinical, clinical, and translational research. Common data elements (CDEs) for certain standard CIBMTR forms have been extracted and associated in the BRIDG model to one of three contexts: Recipient, donor, or stem cell product. Adding HCT content is expected to remove barriers that centers experience in electronic data transfers. HL7 FHIR

In addition to continued curation CDEs in the National Cancer Institute’s Cancer Data Standards Registry and Repository (caDSR) and mapping to BRIDG, the CIBMTR has worked to develop and sustain additional standards for interoperability. Using HL7’s next-generation standards frame work, the CIBMTR created a patient profile and successfully initiated demographic data exchange between a CIBMTR Fast Healthcare Interoperability Resources (FHIR) server and an Epic sandbox

server. In addition to successfully consuming data using FHIR, the CIBMTR deployed a custom operation to evaluate incoming patient demographics and assign a unique recipient ID (CRID) to each patient record. This operation confirms matches between records for the same CRID in both systems and, when no matching CRID is found in CIBMTR systems, automatically establishes one. Once assigned, the CRID will be used by participating centers to provide additional outcomes data, successfully associating the data to the proper patient record.

Disease Risk Index Assignment Tool

In 2015, the CIBMTR launched a DRI Assignment Tool developed by investigators at the Dana Farber Cancer Institute and validated in a large CIBMTR study (Armand et al. Blood, 2014). It is intended for use by clinicians and researchers. The tool was developed for the primary outcome of overall survival after HCT and, at present, only applies to adult patients with hematologic malignancies. It is not intended to give an accurate prognosis for individual patients. In 2018, visitors accessed the DRI Assignment Tool 4,310 times.

VOD Risk Calculator

Launched in October 2017, the VOD Risk Calculator is based on peer-reviewed publication (Strouse C et al. Biology of Blood and Marrow Transplantation, 2018). It is available on the CIBMTR public website and is intended for use by clinicians and researchers. The aim of the VOD Risk Calculator is to identify patients who have a high-risk score for developing VOD, an uncommon, early complication of HCT associated with significant mortality. In 2018, visitors accessed the VOD Risk Calculator 1,226 times.

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.3 ANNUAL MEETINGS

3.3.1 BMT Tandem / TCT Meetings

As of 2019, the BMT Tandem Meetings are called the TCT | Transplantation & Cellular Therapy Meetings of ASBMT and CIBMTR (TCT Meetings) to better reflect an expanded range of interests and activities. The TCT Meetings, held annually in February, include 5 days of scientific sessions and other meetings targeted to worldwide physicians, scientists, and other professionals interested in cellular therapy.

2018 BMT Tandem Meetings

With >3,800 attendees from 48 countries, the 2018 BMT Tandem Meetings included 5 plenary sessions, 9 concurrent sessions, 96 oral abstracts, 2 poster sessions, 7 corporate-supported symposia, and 9 product theaters. Continuing Medical Education (CME) and Continuing Education credits were issued through MCW to physicians and allied health professionals.

Clinical Research Professionals / Data Management Conference With nearly 250 attendees, this conference provided forms and subject matter training, which increases the accuracy with which CIBMTR forms are completed.

BMT CTN Coordinators and Investigators Meetings

With approximately 100 and >400 attendees, respectively, these meetings focused on study management, such as promoting studies and reporting adverse events; procedures, such as enrollment; specific clinical trials; and general cellular therapy subject matters.

Administrative Director Conference With approximately 250 attendees, this conference focused on many topics related to leadership and quality, including methods for improving accuracy and value, staffing models and resilience, and Medicare coverage.

Pharmacists Conference With 250 attendees, this conference presented the latest research and best pharmacy practices with a focus on specific diseases and therapies.

Nursing Conference With 400 attendees, this conference presented the latest research and best nursing practices with a focus on communication techniques and quality of life.

Clinical Education Conference Designed for advanced practice providers, fellows, and junior faculty, and with nearly 300 attendees, this conference focused not only on the latest clinical research but also ethics and resiliency.

Pediatric BMT Program Held in conjunction with the Pediatric Blood and Marrow Transplant Consortium, this meeting is designed for all health professionals involved in caring for children undergoing cellular therapy. With nearly 700 attendees, the meeting shared cutting-edge science and state-of-the-art advances in treatment.

2019 TCT Meetings

The 2019 TCT Meetings are expected to attract approximately 3,800 attendees. The Meetings will include 5 plenary sessions, 9 concurrent sessions, 96 oral abstracts, 1 late breaking abstract session, 2 poster sessions, 8 corporate-supported symposia, and 9

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CIBMTR 2018 Annual Report 3.0 HOW WE SHARE KNOWLEDGE product theaters. 3.3.2 Cellular Therapy Registry Forum

In October 2018, the CIBMTR held a fourth Cellular Therapy Registry Forum. Participants

encompassed 115 attendees representing a variety of industries, including clinical care, research, pharmaceuticals, insurance providers, professional organizations, and government representatives. Topics comprised CAR T toxicity grading criteria, such as cytokine release syndrome and neurotoxicity grading, as well as CAR T toxicity reporting, including an overview of the risk evaluation and mitigation strategy (REMS) program, perspectives of not only centers but also pharmaceutical companies, and development of an effective and streamlined reporting process for CAR T related adverse events. Topics also included cellular therapy center accreditation and data auditing; long-term follow-up infrastructure, including direct patient contact and PRO; and considerations for capturing biospecimens, such as approaches to identify and track predictive biomarkers for immunotherapy response and adverse events in hematologic diseases. Additional presentations consisted of the CMS National Coverage Analysis process and updates from federal agencies.

3.4 DATA MANAGEMENT TRAININGThe CIBMTR has developed comprehensive, secure, and efficient applications to allow centers to electronically submit data to the CIBMTR. Visit the CIBMTR Data Management Training and Reference webpage to access resources.

Data Management Guide Learn about participation in CIBMTR research, center membership, access to FormsNet, data manager education, mentor program, forms submission process and many useful tips and links.

Manuals Find the answers to your data submission questions by accessing the Forms Instructions Manual, which includes general instructions and instructions for each form type.

FormsNet

Learn how to submit data to the CIBMTR via the FormsNet application, a secure clinical research management system, which is in compliance with SCTOD requirements.

Conference Materials Access meeting materials as well as audio and visual presentations on the form submission process presented at Clinical Research Professionals / Data Management Conferences.

AGNIS Learn how to retrieve and transmit form data, extracted directly from your own institution’s database, directly to the FormsNet application using AGNIS, a secure, standards-based system.

Legacy Data Review retired data manuals, forms, and other archived documents for reference purposes and to assist in making changes to legacy data.

Adverse Events Learn how to report adverse events and product issues through FormsNet. Newsletters and eBlasts Read archived issues of the Data Matters Training Newsletter and eBlasts.

Tip Sheets Access sheets providing tips and instructions for various CIBMTR forms.

Online Training Review educational modules developed for new and seasoned data managers. Modules are available on the Online Training webpage.

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Online Trainings New / Updated this Year

APPLICATION SERIES

FormsNet3 Recipient Application Training

DISEASE SPECIFIC SERIES

Lymphoma Pre-/Post-Infusion

INSTITUTIONAL REVIEW BOARD (IRB)

NMDP IRB New Member Orientation

GENERAL TOPICS

Research Sample Submission

DISPONIBLE EN ESPAÑOL

Pre-TED Form 2400

Disease Classification Form 2402

Post-TED Form 2450

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CIBMTR 2018 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA

4.0 HOW WE COLLECT AND MANAGE DATA

4.1 RESEARCH DATA LIFE CYCLEThe Research Data Life Cycle (Figure 4.1) describes the path of data from the point of capture to its ultimate use in analysis, reporting, and publication.

The process begins with data collection. Most centers enter data in FormsNet, a web application now in its third generation. Centers that have implemented local or third-party systems can also capture and submit data electronically using AGNIS. The goal of these applications is to capture high quality data as efficiently as possible.

Following collection, data undergo quality assessment and validation and are extracted and loaded into the CIBMTR Research Database on a monthly basis.

Figure 4.1 Research Data Life Cycle Data Sharing completes the cycle, providing data for analysis that have been collected and curated to ensure research value. These data are extracted from the Research Database in

monthly and quarterly retrievals to serve a range of research and stakeholder needs. The data retrievals provide the basis for research study data files, reports, and externallyrequested datasets. The most commonly used CRF- and TED-level data are also directly available to centers through use of the eDBtC application (Section 3.2.3).

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Abbreviations: AGNIS = A Growable Network Information System, CRID = CIBMTR Recipient Identification Number, CRF = Comprehensive Report Form, FN = FormsNet, IDW = Integrated Data Warehouse, RDB = Research Database


4.2 COLLECTING AND STORING DATA

4.2.1 FormsNet

More than 98% of data collected by the CIBMTR is submitted electronically via FormsNet, a comprehensive electronic data submission system containing >130 forms related to capturing cellular therapy outcomes for donors and recipients. The application was updated in 2018 to provide key enhancements to support operational efficiencies. The CIBMTR also released a number of new forms this year in a continuing effort to maintain alignment between data collection and treatment practices as the cellular therapy field changes and improves. A core set of cellular therapy forms was revised, and new supporting cellular therapy forms were released.

4.2.2 Research Database

The CIBMTR Research Database now contains information on >500,000 patients. Submission of outcomes data is mandatory for allogeneic HCTs in the US and those outside the US that use a US donor; all other submissions

are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and >80% of US autologous HCTs are reported. Cellular therapy outcomes data are also reported to the CIBMTR; since July 2016, >2,000 patients who received cellular therapy were included in the CIBMTR Research Database. Data for approximately 7,500 nonUS patients are collected annually. 4.2.3 Integrated Data Warehouse

Looking to the future, the CIBMTR continues to develop an Integrated Data Warehouse to robustly support future database requirements. The Integrated Data Warehouse uses Oracle's enterprise database and business intelligence infrastructure. The warehouse’s implementation makes use of industry standard data warehousing design patterns in addition to some novel techniques for managing metadata and data quality. The Integrated Data Warehouse is intended to serve as both the aggregation and dissemination point for data collected by the CIBMTR to support its data and research programs as well as external

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data dissemination. The warehouse accommodates the integration of data coming from multiple sources and spanning multiple domains, such as patient outcomes, product data, HLA, biospecimens, and study data.

This year the CIBMTR completed multiple enhancements to support integration of data sources to meet warehouse users’ reporting needs. This integration includes sample data stored in LabVantage system paired with CIBMTR clinical outcomes data, new survey data integration sourced from CIBMTR’s ePRO survey collection system. The CIBMTR also completed a proof of concept extract for the Transplant Center Specific Analysis, in support of advancing the Integrated Data Warehouse. Furthermore, the CIBMTR continued to support cord blood banks with quality assurance processes by providing the cord blood banks additional data around units used for cellular therapy transplants and providing the cord blood banks a secure portal to download their monthly reports (Section 3.2.3).

4.3 ENSURING DATA QUALITY

4.3.1 Continuous Process Improvement

Robust data collection is critical to the success of the CIBMTR. The Continuous Process Improvement (CPI) program ensures timeliness and completeness of data forms submissions (Appendix H).

Recipient Forms

Throughout each trimester, US centers receive CPI reports listing the number of follow-up forms that were due in the previous trimester and the number and percentage of each submitted within the trimester. Letters are sent 3 times per year (January, May, and September) to the Medical Director and Primary Data Manager notifying them if their center met CIBMTR CPI criteria for the trimester. A form is not officially submitted until errors are resolved and all applicable information is submitted and approved. To be compliant, centers must submit ≥90% of forms due for the trimester, for all transplants (autologous and allogeneic, related and unrelated), which occurred since December 3, 2007.

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Donor Forms

The Donor Data Management Team oversees submission of donor work-up and donation forms from NMDP/Be The Match donor, collection, and apheresis centers. Donor CPI reports are generated 4 times per year (January, April, July, and October) for US centers. To be compliant, centers must submit 100% of the forms required for that CPI period. 4.3.2 Verification and

Validation

FormsNet

When data are entered into FormsNet, a series of entry level validation checks takes place to ensure data consistency. This process flags certain errors at the time of entry and allows the CIBMTR to contact the center data manager so errors can be corrected immediately while source documents are readily available. If a data field does not pass the FormsNet validation checks, an error comment is generated, and the data manager is navigated to an error review page to review, resolve, or override the unresolved errors. Lastly, an error report is generated that lists any unresolved errors as well as

errors that have been overridden.

The CIBMTR reacts to data quality issues detected downstream by incorporating additional data quality validations in the upstream data capture system (FormsNet). Detection of issues earlier in the process provides real time feedback to the parties most capable of correcting the problem and improves the overall quality of the data.

Research Database

Data extracted from FormsNet and loaded into the Research Database each month undergo comprehensive validation and verification. These data are rigorously validated for consistency, completeness, and uniqueness using business rules implemented in custom logic for the categories provided below. The Data Quality Team reviews errors and works with centers to correct data.

Rules across various categories are updated and tested as part of ongoing Forms Revision. The CIBMTR continues work to reduce the number of data entry errors. Currently, the rate of form

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rejection due to inconsistently reported data is <2% and has decreased because of recent enhancements in center education as well as enhanced validations built in at the point of entry in FormsNet.

4.3.3 On-Site Data Audit Program

On-going data audits are performed at all CIBMTR participating centers. The audit compares data in source documents maintained at the center with data contained in the CIBMTR Research Database. Clinical Research Associates perform the on-site center audits, spending 3-4 days at each center reviewing original source documents. The overall audit process is displayed in Figure 4.2.

In 2018, 54 centers were scheduled for audit (47 domestic, 7 international). As of December 1, 2018, 40 centers were notified of their final audit results, including requested corrective action follow-up. Of the centers sent reports, 65% passed with ≤3% critical field errors. Of the 14 centers that did not pass the audit, 10

received approval of their corrective action plans, so their audits were determined closed; the remaining 4 centers are in the process of completing requested corrective action.

Consolidation of FACT-CIBMTR Audits

Previously, the CIBMTR and Foundation for the Accreditation of Cellular Therapy (FACT) individually audited data quality at centers. To diminish duplications in the parallel programs and ease the reporting and compliance burdens for centers, the organizations consolidated data audit efforts in 2017. Within this collaboration, the CIBMTR conducts data quality audits and evaluations on behalf of both organizations, and FACT determines whether the results of a data quality audit are satisfactory for accreditation.

All verification of the accuracy of data against source data is done by CIBMTR audit teams on site according to their current practices and schedules. FACT verifies at each annual report and at each application for renewal accreditation the status of CIBMTR data accuracy by

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requiring submission of centers’ most recent CIBMTR audit results for error rates (random errors, systematic errors, and critical field error rates). FACT verifies completeness of data by requiring each center to annually submit the most recent CPI report from the CIBMTR that demonstrates “In Good Standing” related to the on-time submission of completed reports at the rate of at least 90% of

expected. FACT also requires centers to submit internal audits and / or update their corrective action plans annually.

Transplant programs submitting an Annual Report or Renewal Application to FACT are reviewed during the monthly FACT / CIBMTR Data Audit Committee meeting. Successful FACT accreditation will depend on satisfactory completion of this process.

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Figure 4.2 Audit Process

4.4 PROTECTING PATIENTS AND DATA

4.4.1 Human Subjects / HIPAA Compliance

The CIBMTR is committed to the ethical conduct of research. All Coordinating Center personnel maintain Collaborative IRB Training Initiative (CITI) certification.

The NMDP/Be The Match central IRB, which is fully accredited by the Association for the Accreditation of Human Research Protection Programs, reviews all human subject research conducted by the CIBMTR. The CIBMTR maintains compliance with the Health Insurance Portability and Accountability

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Act (HIPAA) Privacy Rule, as applicable. CIBMTR rules requiring the registration of all consecutive HCT recipients ensure the inclusion of women, minorities, and children, so the Research Database population includes women and minorities in the same proportion as found in the general HCT population. Children are included in most CIBMTR studies; their inclusion is dependent on the study focus.

4.4.2 Information Security and Data Privacy

The CIBMTR protects the data and information received from centers and patients. The SCTOD contract requires specific protections through minimum security controls, policies, and standards. The CIBMTR’s data systems are maintained in accordance with the Federal Information Systems Management Act of 2002, and with information security guidance provided by the National Institute of Standards and Technology (NIST 800-53). In accordance with National Institute of Standards and Technology Special Publication 800-18, and in collaboration with HRSA’s Office of Information Technology, the CIBMTR

maintains a System Security Plan that outlines management, operational, and technical controls.From 2008 to 2018, the CIBMTR received from the Chief Information Officer of HRSA an Authority to Operate. Currently the CIBMTR is collaborating with HRSA for renewal of this certification as a modification to the current contract. NMDP/Be The Match also holds an Authority to Operate from HRSA and is also collaborating with HRSA on terms for renewal of this certification as a contract modification. Both organizations maintain a robust information security program, ensure similar standards of information security are applied to all CIBMTR and NMDP/Be The Match systems, and complete annual security control assessments. Controls maintained by the CIBMTR and NMDP/Be The Match, protect the data and information in these systems in ways beyond those required by HIPAA.

In response to changing global personal data protection requirements, the CIBMTR reviewed and updated its Data Use and Processing Policy in 2018. A

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Personal Data Protection Statement is publicly posted on the CIBMTR website along with additional information regarding CIBMTR security infrastructure and how it protects personal data. The CIBMTR understands the importance of its role as a steward of the data it collects and continues to work with domestic and international partners to ensure its systems meet all standards.

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CIBMTR 2018 Annual Report 5.0 WHAT WE WILL DO NEXT

5.0 WHAT WE WILL DO NEXT

In 2019, the CIBMTR will continue to conduct high quality research through a collaborative process. Plans are listed in Tables 5.1-5.3.

Table 5.1 Plans to Enhance Data

Enhance the quality and scope of CIBMTR data

• Revise outcomes data collected to maintain its relevance to the state of the science

• Continue collaborations with investigators and research societies, including the CureSC Consortium, to share data and facilitate studies for patients transplanted for rare, nonmalignant blood diseases

• Provide infrastructure support to global registries

• Continue refinement of the Cellular Therapy Outcomes Registry, including revision of data collection forms and data sharing infrastructure

• Harmonize cellular therapy data collection with global partners

• Expand CPI processes to strengthen consecutive forms submission to the CIBMTR, include international centers, and provide oversight of cellular therapy data collection

• Collaborate with FACT to expand data audit to cellular therapy

• Implement recommendations of the CIBMTR Late Effects Task Force to increase long-term follow-up data collection, resources, and studies.

• Implement electronic collection of PROs as a data resource for clinical investigation

• Enhance data collection systems for risk-based monitoring and lab sample management

• Expand onsite and online data management training and center support

• Improve flexibility of form revision processes and systems

• Enhance data validation and verification processes

• Establish the capability for whole genome sequence analysis of donor-recipient HCT pairs with the goal of identifying markers associated with improved outcomes

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Table 5.2 Plans to Expand Knowledge Sharing

Expand data and knowledge sharing

• In collaboration with ASBMT, host the TCT Meetings to share the latest developments in cellular therapy basic science, translational research, and clinical studies

• Disseminate research findings of key studies to enhance translation of results into clinical practice

• Publish easy to read summaries of key CIBMTR publications for patients, caregivers, and the lay public

• Strengthen relationships with international partners to facilitate data sharing and joint research studies

• Expand regenerative medicine data collection

• In collaboration with the cellular therapy community, evaluate and improve the quality and integrity of data submitted on CTED forms

• Extend features of eDBtC to share cellular therapy data with collaborators and, specifically, international registries

• Develop a tool in Qlikview that will permit sharing of protocol-specific information and delivery of data sets with commercial research partners

• Develop projects that link with administrative payer claims data and other disease and cancer registries to facilitate research on access to, utilization of, and value of cellular therapies

• Continue curation of data elements aligned with accepted standards, including the Cancer Data Standards Registry and Repository, Clinical Data Interchange Standards Consortium (CDISC), and BRIDG model standards, in order to support electronic interoperability

• Develop integration with the Cancer Data Ecosystem and use of HL7/FHIR to integrate with electronic medical records systems and expand automation of data collection

• Complete evaluation of statistical methodology for Center Specific Survival Analysis based on recommendations from the Center Outcomes Forum

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Table 5.3 Plans to Increase Impact

Increase research productivity and scientific impact

• Continue to provide data to CMS for CED studies to allow patients to receive transplants while developing evidence for their efficacy

• Focus effort on studies with highest scientific impact and important clinical and policy implications

• Support Working Committee Chairs to be active leaders in their committees

• Regularly track and share performance metrics as a Working Committee management resource

• Expand use of outcomes data to assist in the design, implementation, and long-term follow-up of clinical trials

• Utilize an ePRO system to support clinical trials, long term follow-up, and other research studies with patients and donors

• Explore opportunities for innovative and important collaborative studies within the Clinical Trials Support and Health Services Research Programs

• Evaluate opportunities to expand the collection and utilization of research samples for HCT and cellular therapy correlative research

• Develop new statistical methodologies

• Enhance data sharing by supporting public access to publication datasets to facilitate collaboration, validation, and expansion of research findings

• Partner with the CureSC and IOTN networks to develop robust data sharing ecosystems

• Continue to partner with professional societies to translate advances in disease risk stratification and transplantation into clinical practice

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CIBMTR 2018 Annual Report 2018 KEY ACCOMPLISHMENTS

2018 KEY ACCOMPLISHMENTS

CIBMTR by the Numbers

RESEARCH DATABASE >400 participating centers >500,000 patients ~25,000 new patients annually

PUBLICATIONS >1,300 publications since inception

109 publications in 2018 74 from Working Committees 13 from BMT CTN 11 from Bioinformatics Research Program 6 from Health Services Research

Program 3 from Statistical Methodology Research

Program 2 from RCI BMT

PRESENTATIONS 76 presentations in 2018 (47 oral and

29 poster) 24 abstracts (14 oral and 10 poster)

presented at the 2018 ASH Annual Meeting

19 abstracts (12 oral and 7 poster) presented at the 2018 BMT Tandem Meetings

6 abstracts (3 oral and 3 poster) presented at the 2018 EBMT Annual Meeting

27 abstracts (18 oral and 9 poster) presented at other national and international conferences

Clinical Outcomes Research Program

WORKING COMMITTEES • 15 committees with >2,700 researchers

worldwide

• 45 global experts voluntarily chair committees

• 195 ongoing studies

• 205 new study proposals; almost 50% were submitted by new investigators

• 46 abstracts (28 oral and 18 poster) presented at national and international conferences

• 74 manuscripts published in peerreviewed journals

SCTOD • Center-Specific Survival Report for

2014-2016 and Transplant Center Volumes Data for 2013-2017 published

• 6th Center Outcomes Forum conducted in September 2018

• 23 study summaries for patients published

CMS CED Studies

• 5 CMS CED studies conducted

• 630 patients received transplants

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More from the

Clinical Outcomes Research Program

CELLULAR THERAPY RESEARCH INITIATIVES • >2,000 patients

>500 received CAR T cell therapy >200 received regenerative medicine

• >200 participating centers

• Cellular Immunotherapy Data Resource funded by NIH

• Interim CIDR Executive Committee established

• 4th Cellular Therapies Forum conducted

• 15-year FDA-mandated follow-up supported for several pharmaceutical companies

• Regenerative Medicine Outcomes Registry strategy meeting hosted

PATIENT-REPORTED OUTCOMES • Pilot project launched to obtain PRO

electronically using the new system

• 28 patients from 2 centers enrolled in the pilot project

INTERNATIONAL INITIATIVES • Scientific Directors shared expertise at

WBMT Workshops in Morocco and China

• Data management staff members collaboratively trained Brazilian data managers at the Brazilian BMT Annual Meeting

Immunobiology Research Program

• Research sample collection and correlative testing for BMT CTN and RCI BMT prospective studies

• 206 centers submitted samples (146 transplant centers, 38 donor centers, and 22 cord blood banks)

• High resolution HLA and KIR typing on 977 related and 3,195 unrelated HCT donor / cord and recipient pairs

• 10,688 samples distributed to investigators for various studies

• 22 publications utilized samples from the Research Repository


RESEARCH REPOSITORY (approximate numbers)

• 3,900 new unrelated recipient samples (64,000 overall)

• 1,300 new related recipient samples (9,500 overall)

• 3,700 new adult unrelated donor samples (71,000 overall)

• 1,200 new related donor samples (9,200 overall)

• 500 new unrelated cord blood samples (12,000 overall)

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Clinical Trials Support Program

BMT CTN • 6 trials completed (40 overall)

• >550 patients accrued to trials (>10,600 overall)

• 6 open protocols

• 8 new protocols in development

• 10,218 new protocol-related biospecimens (408,529 overall)



RCI BMT • 1 new study opened to accrual

(18 overall)

• >2,500 patients accrued (approximately 36,000 overall)

• ePRO system launched to collect outcomes directly from patients

• 8 active studies supported and 4 upcoming studies in development


Health Services Research Program

• Analyzed reimbursement of HCT costs for AML in Medicare beneficiaries

• MDS and AML MATTER: In partnership with ASH, held 2 national summits and a special education session at ASH Annual Meeting to address knowledge gaps among community clinicians

• Focus groups conducted to inform the INSPIRE study, which provides stepped care self-management program for survivors; developed in partnership with Fred Hutchinson Cancer Research Center and Cleveland Clinic, funded by NIH

• Clinician knowledge gaps addressed regarding the diagnosis, risk stratification, and timing of referral for HCT for patients with AML



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Bioinformatics Research Program

• Guidelines for reporting HLA and KIR genotyping via Next Generation Sequencing developed

• Role of genetic ancestry in transplantation investigated

• HLA data from other countries investigated



Research Operations

Statistical Methodology Research Program

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• New statistical models developed

• Statistical integrity of CIBMTR scientific activities ensured

• Articles on cellular therapy-related statistical issues for clinical audiences supported

• Working Committee study investigators supported in developing scientific study protocols using CIBMTR data

• 4 oral abstracts presented at national and international conferences


• 433 data requests fulfilled

• 7 online data management trainings added / updated

• 22 forms (15 revised and 7 new) released in FormsNet

• 19,573 forms for 9,772 patients submitted through AGNIS

• 54 centers (47 domestic and 7 international) audited

• Organizational review and restructure completed

• 2 strategic task forces launched; goals are to transform data strategies and create a build-out plan for commercially funded projects

• Data Use and Processing Policy updated

• 131 SOPs added to the new document management system

• 164 professional development activities completed by CIBMTR staff members

• 620,970 public website page views o 219,983 Data Management Manual o 68,327 Training and Reference

• 30,701 Portal page views o 5,961 DBtC and eDBtC o 4,363 Center Volumes Portal o 3,779 Survival Calculator for

Allogeneic HCT o 687 Data for RFI o 377 Center Performance Analytics o 223 Cord Blood Report Portal*

(*launched this year)

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CIBMTR 2018 Annual Report APPENDIX A1: US CENTERS

APPENDIX A: CENTERS

The CIBMTR represents a network of >400 participating centers in >40 countries that submit outcomes-related data for patients.

APPENDIX A1: US CENTERS

The following table lists the US-based centers that submited data to the CIBMTR Research Database

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Participating Center City State MUD RELATED AUTO

University of Alabama at Birmingham Birmingham AL CRF CRF CRF

Huntsville Hospital Huntsville AL N/A N/A TED

Providence Hospital HPC Transplant Center Mobile AL N/A N/A TED

University of Arkansas for Medical Sciences Little Rock AR CRF TED TED

Banner MD Anderson Cancer Center Gilbert AZ N/A CRF CRF

Banner Blood and Marrow Transplant Program Phoenix AZ CRF CRF CRF

Mayo Clinic Arizona and Phoenix Children's Hospital Phoenix AZ CRF CRF CRF

Cancer Transplant Institute at Virginia G. Piper Cancer Center

Scottsdale AZ CRF CRF CRF

Banner University Medical Center - Tucson Tucson AZ CRF TED TED

Alta Bates Medical Center Berkeley CA TED TED TED

City of Hope National Medical Center Duarte CA CRF TED TED

Scripps Blood & Marrow Transplant Program La Jolla CA CRF CRF CRF

University of California, San Diego Medical Center La Jolla CA CRF CRF CRF

Loma Linda University Cancer Center Loma Linda CA CRF CRF CRF

Cedars-Sinai Medical Center Los Angeles CA CRF TED N/A


for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED level captures basic data, and CRF level captures more detail.

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Children's Hospital of Los Angeles Los Angeles CA CRF TED TED

UCLA Hematologic Malignancy / Stem Cell Transplantation Program

Los Angeles CA TED TED TED

USC BMT Program Los Angeles CA CRF CRF CRF

UCSF Benioff Children's Hospital - Oakland Oakland CA CRF CRF CRF

Children's Hospital of Orange County Orange CA CRF CRF CRF

University of California Irvine Medical Center Orange CA CRF CRF CRF

Lucile Packard Children's Hospital Palo Alto CA CRF CRF TED

Sutter Cancer Center Sacramento CA TED TED TED

University of California-Davis Cancer Center Sacramento CA CRF TED TED

Rady Children's Hospital San Diego San Diego CA CRF CRF CRF

University of California San Francisco Medical Center - Adults

San Francisco CA CRF CRF CRF

University of California San Francisco Medical Center - Pediatrics

San Francisco CA CRF CRF CRF

Stanford University Medical Center Stanford CA CRF CRF TED

The Children's Hospital of Denver Aurora CO CRF CRF CRF

University of Colorado Hospital Aurora CO CRF TED N/A

Colorado Blood Cancer Institute Denver CO CRF TED TED

Yale New Haven Hospital New Haven CT CRF TED TED

Children's National Medical Center Washington DC CRF TED TED

MedStar Georgetown University Hospital Washington DC CRF CRF CRF

Christiana Care Newark DE CRF CRF CRF

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Alfred I. duPont Hospital for Children Wilmington DE CRF CRF CRF


Shands HealthCare & University of Florida Gainesville FL CRF CRF CRF

BMT Program of May Clinic / Nemours and Wolfson Children's Hospital Jacksonville FL CRF CRF CRF

Mayo Clinic Florida - Jacksonville Jacksonville FL CRF CRF CRF

Baptist Hospital of Miami Miami FL N/A N/A CRF

Nicklaus Children's Hospital Miami FL CRF TED TED

University of Miami - Adults Miami FL CRF CRF CRF

University of Miami / Jackson Memorial Hospital Miami FL CRF TED TED

Blood & Marrow Transplant Center, Florida Hospital Medical Group

Orlando FL CRF CRF CRF

Florida Center for Pediatric Cellular Therapy Orlando FL TED TED TED

Memorial Cancer Institute Pembroke

Pines FL TED TED TED

Johns Hopkins All Children's Hospital St. Petersburg FL CRF CRF CRF

H. Lee Moffitt Cancer Center Tampa FL CRF TED TED

Children's Healthcare of Atlanta at Egleston Atlanta GA CRF CRF CRF

Emory University Atlanta GA CRF CRF CRF

The Blood and Marrow Transplant Program at Northside Hospital Atlanta GA CRF CRF CRF

Georgia Cancer Center at Augusta University Health

Augusta GA CRF CRF CRF

Cancer Treatment Centers of America - Southeastern Regional Medical Center

Newnan GA CRF CRF CRF

Kapi'olani Medical Center for Women and Children

Honolulu HI CRF CRF CRF

University of Iowa Hospitals & Clinics Iowa City IA CRF CRF CRF

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St. Luke's Mountain States Tumor Institute Boise ID CRF CRF CRF

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago IL CRF CRF CRF


Comer Children's Hospital / University of Chicago Medicine


Northwestern Memorial Hospital Chicago IL CRF CRF TED

Northwestern University - Department of Immunotherapy Chicago IL CRF TED N/A

The Coleman Foundation Blood and Marrow Transplant Center, Rush University


University of Chicago Medicine Chicago IL CRF CRF CRF

University of Illinois at Chicago Medical Center Chicago IL CRF CRF CRF

NorthShore University HealthSystem Evanston IL N/A N/A CRF

Loyola University Medical Center Maywood IL CRF CRF CRF

Advocate Lutheran General Hospital Park Ridge IL CRF CRF CRF

Cancer Treatment Centers of America Zion IL CRF CRF CRF

Indiana Blood & Marrow Transplantation Indianapolis IN CRF CRF CRF

Indiana University Hospital / Riley Hospital for Children

Indianapolis IN CRF CRF TED

St Vincent Hospital Indianapolis Indianapolis IN N/A CRF CRF

University of Kansas Kansas City KS CRF CRF CRF

Via Christi Hospitals Wichita Wichita KS N/A CRF CRF

University of Kentucky Medical Center Lexington KY CRF TED TED

University of Louisville Hospital / James Brown Cancer Center

Louisville KY CRF TED TED

Louisiana State University Children's Hospital New Orleans LA CRF CRF CRF

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Ochsner Medical Center New Orleans LA CRF CRF TED

Tulane University Medical Center New Orleans LA CRF CRF CRF

Ochsner Louisiana State University Health - Shreveport

Shreveport LA TED TED TED


Beth Israel Deaconess Medical Center Boston MA CRF TED TED

Boston Medical Center Boston MA N/A N/A TED

Dana Farber Cancer Institute & Boston Children’s Hospital Boston MA CRF CRF TED

Dana Farber Cancer Institute at Brigham and Women’s Hospital - Adults

Boston MA CRF CRF TED

Massachusetts General Hospital Boston MA CRF TED TED

Tufts New England Medical Center Boston MA CRF TED TED

Lahey Clinic Medical Center Burlington MA N/A N/A TED

UMass Memorial Medical Center Worcester MA CRF CRF CRF

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore MD TED TED TED

University of Maryland School of Medicine Baltimore MD CRF CRF CRF

National Heart Lung & Blood Institute Bethesda MD N/A TED N/A

National Institutes of Allergy & Infectious Diseases

Bethesda MD N/A TED N/A

National Institutes of Health Bethesda MD N/A CRF N/A

NIH-NCI Experimental Transplantation and Immunology Branch

Bethesda MD N/A CRF CRF

NIH-NCI Matched Unrelated Donor Program Bethesda MD CRF N/A N/A

The University of Michigan Ann Arbor MI CRF TED TED

Children's Hospital of Michigan Detroit MI CRF CRF CRF

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Henry Ford Hospital Bone Marrow Transplant Program

Detroit MI CRF CRF CRF

Karmanos Cancer Institute Detroit MI CRF CRF CRF

Helen DeVos Children's Hospital Grand Rapids MI CRF CRF CRF

Spectrum Health Grand Rapids MI CRF CRF CRF


Masonic Cancer Center University of Minnesota Minneapolis MN CRF CRF CRF

University of Minnesota Blood and Marrow Transplant Program - Adults

Minneapolis MN CRF CRF CRF

University of Minnesota Blood and Marrow Transplant Program - Pediatrics

Minneapolis MN CRF CRF CRF

Mayo Clinic Rochester Rochester MN CRF CRF CRF

Children's Mercy Hospital Kansas City MO CRF CRF CRF

Cardinal Glennon Children's Hospital St. Louis MO CRF CRF CRF

SSM Health Saint Louis University Hospital St. Louis MO CRF TED TED

Washington University School of Medicine St. Louis MO CRF CRF TED

Washington University / St. Louis Children's Hospital St. Louis MO CRF CRF CRF

University of Mississippi Medical Center - Jackson

Jackson MS CRF TED TED

Billings Clinic Cancer Center Billings MT N/A N/A TED

University of North Carolina Hospitals - Chapel Hill Chapel Hill NC CRF CRF CRF

BMT Program at Levine Children's Hospital / Carolinas Medical Center

Charlotte NC CRF CRF CRF

Carolinas Medical Center Blumenthal Cancer Center Charlotte NC N/A CRF CRF

Levine Cancer Institute Charlotte NC CRF CRF CRF

Duke University Medical Center - Adults Durham NC CRF TED TED

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Duke University Medical Center; Pediatric Blood and Marrow Transplant

Durham NC CRF CRF CRF

Wake Forest Baptist Health Winston-Salem NC CRF TED TED

Nebraska Methodist Hospital Omaha NE N/A N/A TED

University of Nebraska Medical Center Omaha NE CRF CRF CRF

Dartmouth-Hitchcock Medical Center Lebanon NH CRF CRF CRF


Hackensack University Medical Center Hackensack NJ CRF CRF CRF

Cancer Institute of New Jersey New Brunswick NJ CRF CRF CRF

UNM Comprehensive Cancer Center Albuquerque NM N/A N/A TED

Children's Hospital at Montefiore Bronx NY CRF TED TED

Montefiore Medical Center Bronx NY TED TED TED

Roswell Park Cancer Institute Buffalo NY CRF CRF CRF

Westchester Medical Center Hawthorne NY CRF CRF CRF

North Shore University Hospital Lake Success NY CRF TED TED

Steven and Alexandra Cohen Children's Medical Center of New York

New Hyde Park NY CRF CRF CRF

Memorial Sloan Kettering Cancer Center - Adults New York NY CRF TED TED

Memorial Sloan Kettering Cancer Center - Pediatrics

New York NY CRF TED TED

Morgan Stanley Children's Hospital of New York New York NY CRF CRF CRF

Mount Sinai Medical Center - New York New York NY CRF CRF TED

New York Presbyterian Hospital New York NY CRF CRF CRF

New York Presbyterian Hospital / Columbia University Medical Center New York NY CRF CRF CRF

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New York University Medical Center New York NY CRF CRF CRF

University of Rochester Medical Center Rochester NY CRF CRF CRF

Stony Brook University Hospital Stony Brook NY TED TED CRF

State University of NY Upstate Medical University

Syracuse NY N/A CRF CRF

Akron Children's Hospital Akron OH CRF CRF CRF

Cincinnati Children's Hospital Medical Center Cincinnati OH CRF CRF CRF


Jewish Hospital Blood and Marrow Transplant Center

Cincinnati OH CRF CRF CRF

University of Cincinnati Medical Center Cincinnati OH CRF CRF CRF

Cleveland Clinic Cleveland OH CRF CRF CRF

Seidman Cancer Center - University Hospitals Cleveland Medical Center

Cleveland OH CRF CRF CRF

Nationwide Children's Hospital Columbus OH CRF CRF CRF

The Ohio State University Medical Center Columbus OH CRF CRF CRF

Oklahoma University Medical Center Oklahoma City OK CRF CRF CRF

Saint Francis Hospital - Oklahoma Tulsa OK N/A TED TED

Legacy Good Samaritan Hospital and Medical Center

Portland OR N/A N/A CRF

Oregon Health and Science University Portland OR CRF CRF CRF

Pediatric BMT Program, Doernbecher Children's Hospital, OHSU

Portland OR CRF CRF CRF

Providence Portland Medical Center Portland OR N/A N/A CRF

Geisinger Medical Center Danville PA CRF TED TED

Penn State Hershey Medical Center Hershey PA CRF CRF CRF

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Abramson Cancer Center University of Pennsylvania Medical Center

Philadelphia PA CRF CRF CRF

Eastern Regional Medical Center Philadelphia PA N/A CRF CRF

Fox Chase Temple University Hospital Bone Marrow Transplant Program

Philadelphia PA CRF TED TED

Hahnemann University Hospital Philadelphia PA CRF TED TED

Philadelphia Children's Hospital Philadelphia PA TED TED N/A

St. Christopher's Hospital for Children Philadelphia PA TED TED TED

Thomas Jefferson University Philadelphia PA TED TED TED


University of Pittsburgh Medical Center Pittsburgh PA CRF CRF N/A

UPMC Children's Hospital of Pittsburgh Pittsburgh PA CRF CRF CRF

West Penn Hospital Pittsburgh PA CRF CRF CRF

Roger Williams Medical Center Providence RI CRF TED TED

Charleston Hematology Oncology Charleston SC N/A CRF CRF

Medical University of South Carolina Charleston SC CRF TED TED

GHS Cancer Institute Greenville SC CRF CRF CRF

Saint Francis Hospital - Greenville Greenville SC CRF CRF CRF

Avera McKennan Transplant Institute Sioux Falls SD CRF CRF CRF

Baptist Blood and Marrow Transplant Memphis TN TED TED TED

St. Jude Children's Research Hospital Memphis TN CRF TED N/A

West Cancer Center / Methodist Healthcare Blood and Marrow Transplant Center Memphis TN CRF CRF CRF

Sarah Cannon BMT Center at Centennial Medical Center

Nashville TN CRF TED TED

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VA Tennessee Valley HCS HSCT Program Nashville Nashville TN TED TED N/A

Vanderbilt University Nashville TN CRF TED TED

Vanderbilt University Veterans Center Nashville TN TED TED N/A

Baylor University Medical Center Dallas TX CRF CRF CRF

Children's Medical Center - Dallas Dallas TX CRF CRF CRF

Medical City Dallas Hospital Dallas TX CRF CRF CRF

UT Southwestern Medical Center - BMT Program Dallas TX CRF CRF CRF

Wilford Hall Medical Center Fort Sam Houston

TX TED TED TED


Cook Children's Medical Center Fort Worth TX CRF CRF CRF

Baylor College of Medicine Center for Cell and Gene Therapy

Houston TX CRF CRF CRF

MD Anderson Cancer Center Houston TX CRF CRF CRF

Covenant Health System Hematopoietic Transplant Program

Lubbock TX N/A TED TED

South Texas Veterans Health Care System San Antonio TX N/A CRF CRF

Texas Transplant Institute San Antonio TX CRF CRF CRF

The Children's Hospital of San Antonio San Antonio TX CRF CRF CRF

Scott and White Memorial Hospital Temple TX N/A N/A CRF

Latter Day Saints Hospital Salt Lake City UT CRF CRF CRF

Utah Blood and Marrow Transplant Program - Adults

Salt Lake City UT CRF CRF CRF

Utah Blood and Marrow Transplant Program - Pediatrics

Salt Lake City UT CRF CRF CRF

University of Virginia Health System Charlottesville VA CRF CRF CRF

Page | 88


Virginia Oncology Associates Norfolk VA N/A N/A TED

Virginia Commonwealth University Massey Cancer Center BMT Program

Richmond VA CRF CRF CRF

University of Vermont Medical Center Burlington VT N/A N/A TED

Fred Hutchinson Cancer Research Center Seattle WA CRF CRF CRF

VA Puget Sound Health Care System Seattle WA TED TED N/A

University of Wisconsin Hospital and Clinics Madison WI CRF CRF CRF

Marshfield Clinic Marshfield WI N/A N/A CRF

Aurora St. Luke's Medical Center Milwaukee WI N/A N/A CRF

Children's Hospital of Wisconsin Milwaukee WI CRF CRF CRF


Froedtert & Medical College of Wisconsin Milwaukee WI CRF CRF TED

West Virginia University Hospitals, Inc. Morgantown WV CRF CRF CRF

Page | 89

CIBMTR 2018 Annual Report APPENDIX A2: INTERNATIONAL CENTERS

APPENDIX A2: INTERNATIONAL CENTERS

The following table lists the international centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: Centers submit data at two levels: TED level captures basic data, and CRF level captures more detail. MED-A forms are administered by EBMT and equivalent to CIBMTR TED forms.

Participating Center City Country MUD RELATED AUTO

Hospital Universitario Austral Buenos Aires Argentina CRF CRF CRF

Institutos Medicos Antartidica Buenos Aires Argentina CRF CRF CRF

Sanatorio Anchorena Buenos Aires Argentina N/A CRF CRF

Hospital Privado de Cordoba Cordoba Argentina CRF CRF CRF

Royal Adelaide Hospital Adelaide Australia CRF CRF N/A

Royal Prince Alfred Hospital Camperdown Australia CRF CRF TED

St. Vincent's Hospital Darlinghurst Australia TED TED N/A

Austin Health Heidelberg Australia CRF CRF N/A

Royal Brisbane & Women's Hospital Herston Australia CRF CRF N/A

Alfred Hospital Melbourne Australia TED TED TED

Royal Children's Hospital, Melbourne Melbourne Australia CRF CRF N/A

Fiona Stanley Hospital Perth Australia CRF CRF N/A

Princess Margaret Hospital for Children Perth Australia CRF CRF TED

Sydney Children's Hospital Randwick Australia CRF CRF TED

Lady Cilento Children's Hospital South

Brisbane Australia CRF CRF CRF

Royal North Shore Hospital St. Leonards Australia TED TED N/A

Page | 90


Royal Melbourne Hospital Victoria Australia CRF CRF N/A


Children's Hospital at Westmead Westmead Australia CRF CRF TED

Westmead Hospital Westmead Australia CRF CRF N/A

Medical University of Vienna Vienna Austria TED TED N/A

Hospital Az Sint-Jan Brugge Belgium TED TED N/A

Children’s University Hospital Bruxelles Belgium TED TED TED

University Hospital Antwerp Edegem Belgium TED TED N/A

University Hospital Gasthuisberg Leuven Belgium TED TED MED-A

Centre Hospitalier Universitaire Department of Hematology

Liege Belgium MED-A MED-A MED-A

Instituto de Cardiologia do Distrito Federal - Unidade de TMO Pietro Albuquerque

Brasilia Brazil CRF CRF CRF

University Estadual De Campinas Campinas Brazil CRF CRF CRF

Hospital de Clínicas Curitiba Curitiba Brazil CRF CRF CRF

Federal University of Ceara Fortaleza Brazil N/A N/A CRF

Hospital Amaral Carvalho Jau Brazil CRF CRF CRF

Hospital de Porto Alegre Porto Alegre Brazil TED TED TED

Instituto Nacional de Câncer Rio de Janeiro Brazil CRF CRF CRF

Albert Einstein Hospital São Paolo Brazil CRF CRF CRF

Bio Sana's São Camilo São Paolo Brazil CRF CRF CRF

Hospital Samaritano São Paulo Brazil CRF CRF CRF

Hospital São Camilo São Paulo Brazil TED TED TED

Page | 91


Hospital Sírio Libanês São Paolo Brazil CRF CRF CRF


Instituto Brasileiro de Controle do Cancer São Paolo Brazil CRF CRF CRF

Instituto da Criança - Universidade de São Paulo

São Paolo Brazil N/A TED TED

Instituto de Oncologia Pediatrica São Paolo Brazil TED TED TED

Real e Benemérita Sociedade de Beneficiência Portuguesa de São Paulo

São Paolo Brazil TED TED TED

Universidad do São Paulo São Paolo Brazil CRF CRF CRF

Alberta Children's Hospital Calgary Canada CRF CRF CRF

Tom Baker Cancer Centre Calgary Canada TED TED TED

Queen Elizabeth II Health Sciences Centre Halifax Canada TED TED TED

Hamilton Health Sciences - Juravinski Hospital and Cancer Centre

Hamilton Canada CRF CRF CRF

Kingston Health Sciences Centre Kingston Canada N/A N/A TED

Centre Hospitalier Universitaire SainteJustine

Montreal Canada CRF CRF CRF

Maisonneuve-Rosemont Hospital Montreal Canada TED TED N/A

McGill University Health Centre - Royal Victoria Hospital Montreal Canada TED TED N/A

Montreal Children's Hospital Montreal Canada TED TED TED

Hotel-Dieu de Quebec Quebec Canada TED TED TED

CHA-Enfant-Jesus Hospital Quebec City Canada TED TED TED

Saint John Regional Hospital Saint John Canada N/A N/A TED

Saskatchewan Cancer Centre Saskatoon Canada TED TED TED

St. John's Health Science Center St. John's Canada N/A N/A TED

Page | 92


Health Sciences North Sudbury Canada N/A N/A TED

Hospital for Sick Children Toronto Canada TED TED TED


Princess Margaret Cancer Center - BMT Program

Toronto Canada TED TED TED

British Columbia's Children's Hospital Vancouver Canada CRF CRF CRF

Vancouver General Hospital Vancouver Canada TED TED TED

CancerCare Manitoba / University of Manitoba

Winnipeg Canada CRF CRF CRF

Pontifica Universidad Catolica de Chile Santiago Chile CRF CRF N/A

Instituto de Trasplante de Médula Ósea de la Costa

Barranquilla Colombia TED TED TED

Clinica de Marly Bogotá Colombia CRF CRF CRF

Charles University Hospital Pilsen Pilsen Czech

Republic CRF CRF CRF

Institute of Hematology & Blood Transfusion

Praha Czech

Republic TED TED N/A

University Hospital Motol Praha Czech


University Hospital, Rigshospitalet Copenhagen Denmark CRF CRF N/A

Children's Cancer Hospital - Egypt Cairo Egypt N/A TED TED

National Cancer Institute Cairo University Cairo Egypt TED TED N/A

Helsinki University Hospital, Comprehensive Cancer Center

Helsinki Finland CRF CRF N/A

Turku University Hospital Turku Finland TED TED N/A

Centre Hospitalier Regional University D'Angers

Angers France TED TED N/A

Hopital Jean Minjoz Besançon France CRF CRF N/A

Hospital A. Michallon, CHU de Grenoble Grenoble France MED-A MED-A N/A

Page | 93


Hopital Claude Huriez, Lille Lille France MED-A MED-A N/A

Institut Paoli-Calmettes Marseille France CRF CRF N/A

Centre Hospitalier Universitaire ARCHET Nice France TED TED MED-A


Hopital Saint Louis Paris France CRF CRF N/A

Centre Hospitalier Lyon Sud Pierre Bénite

Cedex France MED-A MED-A MED-A

Hopital Jean Bernard Poitiers France CRF CRF N/A

Universitaetsklinikum Carl Gustav Carus Dresden Germany CRF CRF CRF

University Hospital of Essen Essen Germany CRF CRF CRF

University Children’s Hospital Frankfurt Frankfurt Germany CRF CRF CRF

Albert-Ludwig University - Freiburg Freiburg Germany MED-A MED-A N/A

UKE Hamburg, Klinik und Poliklinik fuer Stammzelltransplantation

Hamburg Germany CRF CRF N/A

Hannover Medical School Hannover Germany MED-A MED-A N/A

Heidelberg University Clinic Heidelberg Germany TED TED N/A

Christian Albrechts University Kiel Germany CRF CRF CRF

University Leipzig, Bone Marrow Transplant Center

Leipzig Germany CRF N/A N/A

University of Munich Munich Germany CRF CRF N/A

Klinikum Nuernberg, Einheit für Knochenmarktransplantation

Nuernberg Germany MED-A MED-A MED-A

Klinikum der Universitaet Regensburg Regensburg Germany MED-A MED-A N/A

Universitaetsklinikum Tubingen Tubingen Germany CRF TED N/A

University Hospital Tubingen Tubingen Germany TED TED N/A

Page | 94


Universitaetsklinikum Ulm - Adults Ulm Germany CRF CRF TED

Deutsche Klinik fuer Diagnostik - Wiesbaden Wiesbaden Germany CRF CRF N/A

University Hospital of Patras, Patras University Medical School Rio Patras Greece TED TED TED

Chinese University of Hong Kong, Prince of Wales Hospital Shatin Hong Kong CRF CRF N/A


Gujrat Cancer & Research Institute Ahmedabad India CRF CRF CRF

SANKALP-CIMS Centre for Paediatric Bone Marrow Transplantation

Ahmedabad India N/A CRF CRF

People Tree Centre for Paediatric Bone Marrow Transplantation

Bangalore India N/A CRF N/A

Rajiv Gandhi Cancer Institute and Research Center, New Delhi Delhi India CRF CRF CRF

Asian Institute of Medical Sciences, Faridabad

Faridabad India CRF CRF CRF

Apollo Hospital International Ltd. Gandhinagar India N/A TED TED

Medanta - The Medicity Hospital Gurgaon India CRF CRF CRF

Sri Aurobindo Medical College Indore India N/A CRF CRF

South East Asia Institute for Thalassemia Jaipur India N/A CRF N/A

Christian Medical College, Ludhiana Ludhiana India TED TED TED

Jawaharlal Institute of Postgraduate Medical Education and Research

Puducherry India N/A TED TED

Deenanath Mangeshkar Hospital Pune India CRF CRF CRF

Sahyadri Speciality Hospital Pune India CRF CRF CRF

Christian Medical College Hospital Vellore India CRF CRF N/A

Rambam Medical Center Haifa Israel CRF CRF CRF

Hadassah Medical Center Jerusalem Israel CRF CRF N/A

Page | 95


Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital Petah Tikva Israel CRF CRF CRF

Tel-Aviv Sourasky Medical Center Tel-Aviv Israel CRF CRF CRF

Chaim Sheba Medical Center - Pediatrics Tel-Hashomer Israel CRF CRF CRF

Sheba Medical Center Tel-Hashomer Israel CRF CRF N/A

Sant’Orsola-Malpighi Hospital Bologna Italy MED-A MED-A N/A


University Bologna - Pediatrics Bologna Italy TED TED TED

Spedali Civili di Brescia Brescia Italy N/A N/A MED-A

Ospedale Ferrarotto Catania Italy CRF CRF TED

Policlinic Tor Vergata University - Rome Transplant Network

Rome Italy CRF CRF N/A

Universita Cattolica Sacro Cuore Rome Italy TED TED TED

Ospedale Molinette Torino Italy MED-A MED-A N/A

Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

Ciudad de Mexico

Mexico CRF CRF CRF

Instituto Nacional de Pediatria Mexico Coyoacan Mexico CRF CRF CRF

Hospital Universitario Dr. José Eleuterio González, Universidad Autónoma de Nuevo León

Monterrey Mexico CRF CRF CRF

Centro de Hematologia y Medicina Interna Clinica RUIZ de Puebla

Puebla Mexico TED TED TED

VU Medical Center - Amsterdam Amsterdam Netherlands TED TED TED

University Medical Centre Groningen Groningen Netherlands TED N/A N/A

Leiden University Medical Centre Leiden Netherlands CRF TED N/A

Academic Hospital Maastricht Maastricht Netherlands CRF TED N/A

Radboud University Medical Center Nijmegen Netherlands TED TED N/A

Page | 96


Erasmus MC - Daniel Den Hoed Cancer Center

Rotterdam Netherlands TED TED N/A

University Medical Center Utrecht Utrecht Netherlands TED N/A N/A

University Medical Center Utrecht Pediatrics Utrecht Netherlands CRF N/A N/A

Auckland City Hospital Auckland New Zealand CRF CRF N/A

Starship Children's Health Auckland New Zealand CRF CRF N/A


Christchurch Hospital Christchurch New Zealand CRF CRF TED

Wellington Blood and Cancer Centre Wellington New Zealand CRF CRF N/A

Rikshospitalet - The National Hospital Oslo Norway CRF N/A N/A

Shifa International Hospitals Islamabad Pakistan N/A CRF CRF

Armed Forces Bone Marrow Transplant Center

Rawalpindi Pakistan N/A CRF CRF

Hospital Rebagliati Lima Peru CRF CRF CRF

Instituto Nacional Salud Del Niño San Borja San Borja Peru N/A TED N/A

Silesian Medical Academy Katowice Poland CRF CRF N/A

Poznan University of Medical Sciences Poznan Poland MED-A MED-A N/A

Medical University of Warsaw Warsaw Poland CRF CRF N/A

Lower Silesian Center for Cellular Transplantation

Wroclaw Poland CRF CRF N/A

Instituto Portugues de Oncologia - Lisbon Lisbon Portugal CRF CRF N/A

Instituto Portugues de Oncologia - Porto Porto Portugal MED-A MED-A MED-A

King Abdulaziz Medical City - Riyadh Riyadh Saudi Arabia N/A CRF CRF

King Abdullah Specialist Children's Hospital (KASCH) Riyadh Saudi Arabia CRF CRF CRF

Page | 97


King Fahad Medical City Riyadh Saudi Arabia CRF CRF CRF

King Faisal Specialist Hospital & Research Center - Adults

Riyadh Saudi Arabia CRF CRF N/A

King Faisal Specialist Hospital & Research Center - Pediatrics

Riyadh Saudi Arabia CRF CRF N/A

National University Hospital - Adults Singapore Singapore CRF CRF CRF

National University Hospital - Pediatrics Singapore Singapore CRF CRF CRF

Parkway Cancer Centre Singapore Singapore CRF CRF CRF


Singapore General Hospital Singapore Singapore CRF CRF CRF

Slovak Medical University Bratislava Slovak


Samsung Medical Center Seoul South Korea CRF CRF CRF

Seoul St. Mary's Hospital Catholic BMT Center Seoul South Korea CRF CRF CRF

Hospital Infantil Vall d'Hebron Barcelona Spain MED-A MED-A MED-A

University of Barcelona Barcelona Spain TED TED N/A

Institut Català d'Oncologia - IDIBELL L'Hospitalet Spain CRF CRF MED-A

Gregorio Maranon University General Hospital Madrid Spain CRF CRF CRF

Hospital Infantil Universitario Niño Jesús Madrid Spain CRF CRF MED-A

Hospital Puerta Hierro Madrid Spain MED-A MED-A N/A

Son Dureta Hospital Palma

Mallorca Spain CRF CRF N/A

Hospital Universitario La Fe Valencia Spain CRF CRF N/A

Sahlgrenska University Hospital Gothenborg Sweden CRF CRF N/A

University Hospital of Lund Lund Sweden CRF MED-A N/A

Karolinska University Hospital, Stockholm Sweden CRF CRF N/A

Page | 98


Department of Cellular Therapy and Allogeneic Stem Cell Transplantation

University Hospital - Uppsala Uppsala Sweden CRF CRF N/A

Basel Kantonsspital Basel Switzerland CRF CRF N/A

Geneva University Hospitals Geneva Switzerland MED-A MED-A N/A

University Hospital - Zurich Zurich Switzerland MED-A MED-A N/A

King Chulalongkorn Memorial Hospital Bangkok Thailand TED TED TED


Ankara University Faculty of Medicine Ankara Turkey TED TED N/A

Medical Park Hospital - Antalya Antalya Turkey CRF CRF CRF

Birmingham Children's Hospital Birmingham United

Kingdom TED TED N/A

Birmingham Heartlands Hospital Birmingham United

Kingdom TED TED TED

Queen Elizabeth Hospital - Birmingham Birmingham United

Kingdom CRF CRF N/A

Bristol Children's Hospital Bristol United

Kingdom CRF CRF N/A

Addenbrooke's NHS Trust Cambridge United

Kingdom CRF CRF N/A

Western General Hospitals Edinburgh United

Kingdom TED TED TED

Beatson West of Scotland Cancer Centre Glasgow United

Kingdom MED-A MED-A N/A

Royal Hospital for Sick Children Glasgow United

Kingdom CRF CRF N/A

St. James University Hospital Leeds United

Kingdom TED TED N/A

Great Ormond Street Hospital London United

Kingdom CRF CRF N/A

Imperial College London / Hammersmith Hospital London

United Kingdom

CRF CRF N/A

Imperial College - St. Mary's Hospital London United

Kingdom CRF CRF N/A

Haematology & Cancer Services - Newcastle Newcastle

United Kingdom

CRF CRF TED

Page | 99


British Hospital Montevideo Uruguay N/A CRF CRF

Centro de Trasplante del Servicio Medico Integral (SMI) Montevideo Uruguay CRF CRF CRF

Hospital Maciel Montevideo Uruguay CRF CRF CRF

Cuidad Hospitalaria Dr. Enrique Tejera Valencia Venezuela TED TED TED

Page | 100

APPENDIX B: COORDINATING CENTER CIBMTR 2018 Annual Report ORGANIZATIONAL STRUCTURE AND LEADERSHIP

APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP

The CIBMTR Coordinating Center resides on two campuses. One is located at MCW in Milwaukee, WI, and the other is located at NMDP/Be The Match in Minneapolis, MN. The Coordinating Center provides administrative, statistical, data management, clinical trials, IT, and personnel support for CIBMTR activities, and it benefits from a unique, collegial partnership with the Division of Biostatistics of MCW.

CIBMTR Milwaukee has approximately 85 employees and is an academic division of the MCW Department of Medicine. The Milwaukee office receives administrative support from the MCW departments of Grants and Contracts, Development, Public Affairs, Human Resources, and Office of Technology Development as well as the Department of Medicine Administration. CIBMTR

Minneapolis has approximately 100 employees, and several NMDP/Be The Match departments provide support for CIBMTR activities, including IT, Finance, Contracts, and Marketing and Communications.

Page | 101

CIBMTR 2018 Annual Report APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS

Page | 102

CIBMTR 2018 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS

APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS (PART 1)

Page | 103

A WiedmeyerAssistant

AdministrativeR Schulist

DL CampbellAssistant, Sr

AdministrativeR Dunn

Coordinator IIProgram

M PatelData Associate

TBDA PopeT Hunt

K BhavsarCoordinator II

Program

LA LaczkowskiSpecialist III

TrainingS MeiersResearchClinical

Manager,

B Shaw, MD, PhDJD Rizzo, MD, MS

M Pasquini, MD, MSS Lee, MD, MPH2

K Flynn, PhDM Eapen, MD, MS

L Burns, MDSenior Scientific Director

D Weisdorf, MD1Senior Research Advisor

D ConferDirector

Associate Scientific Medical Faculty

W Saber, MD, MS

3M Riches, MD, MSP Hari, MD, MS

M Hamadani, MDA D’Souza, MBBS, MD

1M Arora, MD,MSScientific Director

Medical Faculty

M HembrookSpecialistFinancial S BanagisAssociateFinancial

L LarsonH Fischer

rAssistant, SAdministrative

A CurtissAssociate

ve Administrati

lliams M WirvisorSupe

ve stratiAdmini

E TuschlData Development

Manager, Senior

C AbelAssistant III

Clinical Research S Meyers

Coordinator IClinical Research

A KummerowMetadata Analyst

A PrenticeCoordinator II

Clinical Research A Benoit

Coordinator IIResearch

Study Clinical J Myers

Coordinator IIIClinical Research

M KleinA Hantke

Coordinator IResearch Clinical

C-Grady, PA-J BrunnererationsData OpDirector, Program

YoungS NeunagerMagram Proorate Corp

M AndersonCoordinator II

Program Corporate

J ClassCoordinator I

gram ProA Halfmann

ICoordinator IProgram

BreyM IInator ICoordi

Program BDTcalistSpei

Commu ns nicatioJ Motl

Smith-J GillisMedical Writer

sherS Fis DBusines evelopment

ctor, Dire

spalecP Veger, SrMananess Busi

PhD, MBAP Steinert,Executive Director

M Horowitz, MD, MSChief Scientific Director

MKE Campus

-= CIBMTR

University of North Carolina Hospitals3

Fred Hutchinson Cancer Research Center2

University of Minnesota1


APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS

Page | 104


APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS (PART 2)

= CIBMTR - MKE

Page | 105

CIBMTR 2018 Annual Report Campus

APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 1)

Page | 106

D RusnakC Erickson

K ChristiansonAssistantResearch Clinical

M TierneyB Person

A MussetterN CarlisanoSpecialistResearch Clinical

C JohnsonManager

Clinical Data

A HowardResearch

Immunobiology Supervisor,

TBDBiostatistician

Supervisor,

K Lozano BejaranoC Jacox

L BankoleAssociate

Survey Research

D MattilaSupervisor

GroupSurvey Research

J DworskiCoordinator

Research Prospective

A FoleyProject Manager

BMT CTN

TBDJ VogelB Protz

H KobusingyeA AdamsManager

Clinical Project

E LeckroneResearch

Prospective Director,

S WaldvogelA Spahn

A EricksonScientistResearch

Immunobiology

W WangScientists

Bioinformatics Associated

TBDScientists

Bioinformatics

Y BolonGenomic Services

Supervisor,

P BashyalArchitectSoftware

Bioinformatics D Roe

S FingersonScientists

Bioinformatics A Madbouly

ScientistsBioinformatics

Senior

TBDBioinformatics Research

Senior Manager,

A CarlsonSpecialist

AdministrativeM BrownScientistResearch

Immunobiology Senior

Green-C VierraScientistResearch

Immunobiology Principal

J SeesM Formanek

BiostatisticianC Fretham

P ChitphakdiathaiBiostatistician

Senior

, MBS1S SpellmanDirector, Immunobiology

Steven Devine, MDMedical Director, Research Operations

Senior Vice President and Senior

R King, MPHVice President, CIBMTR Minneapolis

Mary M Horowitz, MD, MSChief Scientific Director


= CIBMTR - MKE Campus

= CIBMTR - MSP Campus

Page | 107



Page | 108

S SorensenK Malmum

AnalystMetadata W ZhangAnalyst

Metadata Senior

R RennerArchitect

Principle Data

R LatchanaCoordinator III

Research Clinical

A DraxlerSuper UserFormsNet

TBDHouse)-Line (In-CRA

Supervisor,

K KaneLine (Field)-CRA

Supervisor,

K ZaffranA YostC Vang

J TuckerJ PetersonK MajureJ Larsen

C KunakomAE Hendrickson

E EklundAssociate

Clinical Research AA Hendrickson

Research AssociateSenior Clinical

E EichSpecialist

Clinical Database L Wendland

M ProueK Cook

J BloomquistA Birch

Project ManagerMonitoring

Clinical

D ChristiansonData Quality Assurance

Compliance & Senior Manager,

K BloomquistData OperationsDirector,

Steven Devine, MDMedical Director, Research Operations

Senior Vice President and Senior

M AmmiCoordinatorOperations

Data

K OlsonJ Hullerman Umar

Clinical Trials AssistantP Wallace

N VoitE Mitchem

AnalystClinical DataA CastellonAssociate

Clinical Research J TenneyAssociate

Senior Data Research

TBDC TurnerS TaskyA Mitch

Coordinator IIClinical Research

S LoganCoordinator III

Program K GardnerTeam Lead

Improvement Process

Continuous

T TholeC Olson

R KrunkkalaCoordinator II

Research Clinical A Ewer

Coordinator IIIResearch Clinical

A HauckCustomer ServiceSupervisor, Center

J VueB Schlicht

P LeeSpecialist I

Data Support T WinderJ KhangL Horne

Specialist IIData Support

K KutznerSpecialist

Quality Control

B LevesqueSupport

Supervisor, Data

J WeberData Capture

Senior Manager,



MSP Campus

- CIBMTR =

ampusMKE C

- CIBMTR =



Page | 109

M McCulloughNMDP CIO

TBDSpecialist

Administrative H Severance

Learning CoordinatorD Lu

SpecialistDevelopment

Learning C Jobe

SpecialistResearch Protection

Senior Human J Tkachenko

IRB -Specialist Research Protection

Senior Human

L SchaeferleAnalyst

Associate Data D Turner

Data AnalystT Wirth

D McDonellAnalyst

Senior Data K Cich

ScientistBioinformatics

E WilliamsScientist

Bioinformatics Senior

C JordahlEngineerSoftware J Smith

R KumarN Hood

EngineerSenior Software

E ZinkArchitect

Principal Data

V RosenthalSpecialist

Administrative

J OakesAnalyst

Quality Assurance B Samba

Assurance AnalystSenior Quality

J SchneiderProgrammer / AnalystSenior Bioinformatics

K SchaperSenior Data Analyst

B MiliusScientist

Principal Research

YarraV Assurance

r, Quality Manage

J BrelsfordAnalyst

Data Storage A Westin

AdministratorSystem C Yang

AdministratorSenior System

hanE C ManagerSolutions

DM CampbellProject Coordinator

S EwerL Clements

ScrumMasterTBD

B WakarukProject Manager

L MobergSystems AnalystSenior Business

S Freeman Manager

Senior Project B Burgess

Business Architect

K GeeanagementProgram M

Senior Manager,

L GabrielsonZ Ahmed

Engineer (BI)Software S Stagg

Engineer (ETL)Senior Software

R FritschArchitect (BI)

Solutions

J P ackollonsutiSol

Mana , Data ger

stegaardM PreMTR IT SystemsDirector, CIB

Steven Devine, MDDirector, Research Operations

Senior Vice President and Senior Medical




= CIBMTR - MKE Campus = CIBMTR - MSP Campus

Page | 110

CIBMTR 2018 Annual Report APPENDIX B3: COORDINATING CENTER LEADERSHIP

APPENDIX B3: COORDINATING CENTER LEADERSHIP

SENIOR LEADERSHIP

Mary M. Horowitz, MD, MS • Chief Scientific Director for the CIBMTR

• Principal Investigator for the Data and Coordinating Center of the BMT CTN

• Research Director for the SCTOD • Robert A. Uihlein Professor of Hematologic Research at MCW

• Associate Director for Cancer Genomics • Attending physician in the MCW HCT program

C. Randy Mills, PhD • Executive Director for the CIBMTR • Chief Executive Officer of NMDP/Be The Match

Steven Devine, MD • Associate Scientific Director for CIBMTR Minneapolis • Senior Vice President and Senior Medical Director of Research

Operations for NMDP/Be The Match • Co-Scientific Director of the RCI BMT

Daniel Weisdorf, MD • Senior Research Advisor for the CIBMTR • Scientific Director of the Acute Leukemia Working Committee • President of the WBMT • Professor of Medicine at the University of Minnesota • Chief of the Division of Hematology, Oncology, and Transplantation

at the University of Minnesota • Associate Chair of Clinical Research in the Department of Medicine

at the University of Minnesota

Page | 111


• Attending physician at the University of Minnesota Medical Program Mei-Jie Zhang, PhD • Chief Statistical Director for the CIBMTR • Biostatistician for the Acute Leukemia and Graft Sources and

Manipulation Working Committees • Professor of Biostatistics at MCW

Page | 112


Mary Eapen, MBBS, MS • Senior Scientific Director of Research Operations for the CIBMTR • Scientific Director for the Graft Sources and Manipulation; Pediatric Cancer; and Primary

Immune Deficiencies, Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committees • Protocol Officer for several BMT CTN trials • Professor of Medicine at MCW

Kathryn E. Flynn, PhD • Senior Scientific Director of Patient-Reported Outcomes for the CIBMTR • Associate Professor of Medicine at MCW

J. Douglas Rizzo, MD, MS • Senior Scientific Director and Principal Investigator of the SCTOD for the CIBMTR • Professor of Medicine at MCW • Associate Director of Clinical Operations for the Froedtert and MCW Cancer Center • Attending physician in the MCW HCT program

Page | 113


Bronwen Shaw MD, PhD • Senior Scientific Director of Data Operations for the CIBMTR • Co-Scientific Director of the RCI BMT • Scientific Director of the Late Effects and Quality of Life Working Committee as well as the Donor Health and Safety Working Committee • Professor of Medicine at MCW • Attending physician in the MCW HCT program

Linda Burns, MD • Vice President and Senior Scientific Director of the Health Services Research Program for the

CIBMTR • Co-Scientific Director of the RCI BMT • Medical Director, Medical Services, NMDP/Be The Match • Past President of ASH, 2014

Page | 114


Marcelo Pasquini, MD, MS • Senior Scientific Director of CIBMTR Clinical Trials Support – BMT CTN for the CIBMTR • Scientific Director for the Autoimmune Diseases and Cellular Therapies Working Committee

and the Regimen-Related Toxicity and Supportive Care Working Committee • Protocol Officer and Director of Medical Monitors for the BMT CTN • CIBMTR Representative to the WBMT • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program

Martin Maiers, MS • Vice President of Biomedical Informatics for the CIBMTR • Staff liaison to the NMDP/Be The Match Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee) • Co-Chair of Informatics: International Histocompatibility and Immunogenetics Workshop • Member of World Health Organization HLA Nomenclature

Committee

Roberta King, MPH • Vice President for CIBMTR Minneapolis • Oversees the administrative, research administration, scientific,

and statistical support activities of CIBMTR Minneapolis • Directs research operations functional teams for human subject

protection program, data management, auditing and monitoring, observational research, and prospective research

• Staff Liaison to the NMDP/Be The Match Donor and Patient Safety Monitoring Advisory Group

Patricia Steinert, PhD, MBA • Executive Director for CIBMTR Milwaukee • Oversees administrative, scientific, and statistical activities of CIBMTR Milwaukee • Directs research operations functional teams for data operations, statistical operations, IT, and

advancement

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SCIENTIFIC DIRECTORS SCIENTIFIC DIRECTORS SCIENTIFIC DIRECTORS


Mukta Arora, MD, MBBS, MS • Scientific Director of the Graft-versus-Host Disease Working

Committee • Associate Professor of Medicine at the University of Minnesota • Attending physician in the University of Minnesota HCT program

Anita D’Souza, MD • Assistant Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors Working

Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program

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Parameswaran Hari, MD, MS • Scientific Director of the Plasma Cell Disorders and Adult Solid

Tumors Working Committee • Armand J. Quick – William F. Stapp Professor of Hematology at MCW • Associate Division Chief, Hematology and Oncology at MCW • Attending physician in the MCW HCT program

Mehdi Hamadani, MD • Scientific Director of the Lymphoma Working Committee • Director of Adult Blood and Marrow Transplantation Program at MCW • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program

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Stephanie J. Lee, MD, MPH • Senior Scientific Director of Immunobiology Research for the CIBMTR • Co-Scientific Director of the Immunobiology Working Committee • Professor of Medicine at the University of Washington • Attending physician member in the Fred Hutchinson Cancer Research Center HCT program

Marcie Riches, MD, MS • Scientific Director of the Infection and Immune Reconstitution Working Committee • Protocol Officer and Medical Monitor for several BMT CTN trials • Clinical Associate Professor of Medicine at the University of North Carolina at Chapel Hill • Director of BMT Clinical Research and Data Quality at the University of North Carolina

at Chapel Hill • Attending physician in the University of North Carolina Lineberger Comprehensive Cancer

Center HCT program Wael Saber, MD, MS • Scientific Director of the Chronic Leukemia and Health Services

and International Studies Working Committees • Assistant Scientific Director of the Acute Leukemia Working

Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program

Stephen Spellman, MBS • Co-Scientific Director of the Graft-versus-Host Disease and Immunobiology Working

Committees • Director of Immunobiology Research for the CIBMTR

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• Principal Investigator for the Research Repository • Staff liaison to the NMDP/Be The Match Cord Blood Advisory Group Research subcommittee

and Histocompatibility Advisory Group (also serves as the CIBMTR Immunobiology Steering Committee)

• Program Manager for the NMDP/Be The Match Office of Naval Research Grant

• Graduate faculty in the University of Minnesota Bioinformatics and Computational Biology program

STATISTICAL DIRECTORS

Kwang Woo Ahn, PhD • Biostatistician for the Lymphoma and Pediatric Cancer Working

Committees • Associate Professor of Biostatistics at MCW

Ruta Brazauskas, PhD • Biostatistician for the Autoimmune Diseases and Cellular Therapies, Health Services and

International Studies, and Late Effects and Quality of Life Working Committees • Associate Professor of Biostatistics at MCW

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Raphael Fraser, PhD • Biostatistician for the Plasma Cell Disorders Working Committees

and the BMT CTN • Assistant Professor of Biostatistics at MCW

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Soyoung Kim, PhD • Biostatistician for the Infection and Immune Deficiencies and the Primary Immune Deficiencies,

Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committees • Assistant Professor of Biostatistics at MCW

Ying Liu, PhD • Biostatistician for the Chronic Leukemia and Graft-versus-Host Disease Working Committees • Assistant Professor of Biostatistics at MCW

Brent Logan, PhD • Biostatistician for the Donor Health and Safety and the RegimenRelated Toxicity and Supportive

Care Working Committees • Lead Statistician for the BMT CTN and Statistical Consultant to the NMDP/Be The Match • Professor of Biostatistics at MCW • Director of the Division of Biostatistics at MCW

Tao Wang, PhD • Biostatistician for the Graft-versus-Host Disease and Immunobiology Working Committees • Associate Professor of Biostatistics at MCW

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OTHER LEADERSHIP STAFF

Erik Bergman, MBA, MS • Director of IT for the CIBMTR in Milwaukee • Leads the IT staff in Milwaukee, which is organized in four teams: Database, Applications Development, Technology Services, and Project Management • Oversees management of the Research Database, including

extraction of data from source systems as well as their transformation and loading to the database

• Responsible for data retrievals from the Research Database as well as key solutions for sharing data with stakeholders

Janet Brunner-Grady, PA-C • Program Director of Data Operations for the CIBMTR • Manages the clinical research coordinators, develops training

programs, and monitors center CPI • Assists clinical research coordinators on both campuses with clinical

transplant-related questions

Kristina Bloomquist • Director of Data Operations for CIBMTR Minneapolis • Oversees data capture (metadata, forms revision and FormsNet application testing and

development) as well as compliance and quality assurance (data quality, auditing and monitoring) teams Sharniece Covill

• Director of Business Operations for the CIBMTR • Oversees the deliverables and milestones associated with the full

portfolio of CIBMTR projects

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Sherry Fisher • Director of Business Development for the CIBMTR • Manages the advancement activities and the Corporate Program, which generates new revenue

to create a continued source of nonfederal financial support • Oversees meetings and communications activities, including the annual TCT Meetings

Erin Leckrone

• Director of Prospective Research for the CIBMTR • Manages the activities of the RCI BMT, including the Survey Research Group • Oversees the administration of the Clinical Trials Advisory Committee

Waleska S. Pérez, MPH • Program Director of Statistical Operations and Clinical Outcomes Research for the CIBMTR • Oversees Master’s-level statisticians • Provides administrative oversight of the Clinical Outcomes Research Program

Matt Prestegaard • Director of IT for the CIBMTR in Minneapolis • Focuses on developing IT systems in the areas of searching and matching, case management,

international communications, and research • Former Chair of the European Marrow Donor Information System Technical Group • Former Chair of the World Marrow Donor Association IT Working Group

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CIBMTR 2018 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP

APPENDIX C: COMMITTEE MEMBERSHIP

CIBMTR committees provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of its scientific and medical communities. All committees and their functions are listed in Table 1.4.

APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP

The Advisory Committee provides oversight for CIBMTR policies and scientific agenda and also partners with the Working Committees to prioritize scientific studies. Members are elected to three-year terms by the CIBMTR Assembly and must be from qualifying CRF centers. All Advisory Committee terms begin on March 1.

ELECTED MEMBERS

Chair Rob Soiffer, MD, Dana Farber Cancer Institute, Boston, MA

Past Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

VICE CHAIRS

North America Paul Carpenter, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Europe Katarina Le Blanc, MD, PhD, Karolinska University Hospital, Centre for Allogeneic Stem Cell Transplantation, Stockholm, Sweden

Central / South America Nelson Hamerschlak, MD, PhD, Albert Einstein Hospital, Sao Paolo, Brazil

Asia / Africa / Australia Biju George, MBBS, MD, Christian Medical College Hospital, Vellore, India

MEMBERS AT LARGE North America Karen Ballen, MD, Massachusetts General Hospitals, Boston, MA

Michael Bishop, MD, University of Chicago Medicine, Chicago, IL

Claudio Brunstein, MD, PhD, University of Minnesota Blood and Marrow Transplant Program, Minneapolis, MN

Navneet Majhail, MD, MS, Cleveland Clinic Foundation, Cleveland, OH

Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center, New York, NY

Bart Scott, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Non-North America Jane Apperley, MBChB, MD, FRCP, Imperial College / Hammersmith Hospital, London, United Kingdom

Hildegard Greinix, MD, Medical University of Vienna, Vienna, Austria

Shahrukh Hashmi, MD, MPH, King Faisal Specialist Hospital & Research

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Center, Riyadh, Saudi Arabia Tracey O’Brien, MBA, LLM, MBChB, BSc, Sydney Children's Hospital, Sydney, Australia

Anna Sureda, MD, PhD, Institut Català d'Oncologia-Hospital Duran I Reynals, Barcelona, Spain

Jeffrey Szer, MD, Royal Melbourne Hospital City Campus, Victoria, Australia

APPOINTED MEMBERS ASBMT Representative John DiPersio, MD, PhD, Washington University School of Medicine, St. Louis,

MO

Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA

Business Representative Jeff Chell, MD, NMDP/Be The Match, Minneapolis, MN

Collection Center Representative James Mason, MD, Scripps Blood and Marrow Transplant Program, San Antonio, TX

Cord Blood Bank Representative Andromachi Scaradavou, MD, New York Blood Center, Long Island City, NY

Donor Center Representative TBD

Patient / Family Representatives Jeffrey Haertling, Tierra Verde, FL

(Co-Chairs, Consumer Advocacy Committee)

EX OFFICIO MEMBERS

Hillary Hall, Cambridge, MA

Executive Director Randy Mills, PhD, NMDP/Be The Match, Minneapolis, MN

Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI

Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI

Senior Scientific Director SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI

Associate Scientific Director CIBMTR Minneapolis Steven Devine, MD, CIBMTR, Minneapolis, MN

Vice President CIBMTR Minneapolis Roberta King, MPH, CIBMTR, Minneapolis, MN

Senior Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI

Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN

Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN

NMDP/Be the Match / MCW / HRSA Contracting Officer Representative Nawraz Shawir, MBBS

NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)

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MCW / HRSA Contracting Officer Representatives

Janet Kuramoto-Crawford, PhD, MHS Marilyn Levi, MD

MCW / NCI Project Officer William Merritt, PhD, Bethesda, MD

MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD Nahed El Kassar, MD, PhD, Bethesda, MD

MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD

Nominating Committee Chair Corey Cutler, MD, MPH, Dana Farber Cancer Institute, Boston MA

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CIBMTR 2018 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

The Executive Committee, a subcommittee of the Advisory Committee, ensures that the organization carries out its mission and adheres to CIBMTR policies and procedures. It also provides advice and counsel to the Coordinating Center between meetings of the Advisory Committee. Specifically, the Executive Committee is responsible for providing scientific and policy advice to the Chief Scientific Director and Coordinating Center, reviewing audit results and making recommendations for improvement, and appointing a CIBMTR Co-Chair and additional CIBMTR representatives to the scientific organizing committee for the annual TCT Meetings. All Executive Committee terms begin on March 1.

ELECTED MEMBERS

Chair Rob Soiffer, MD, Dana Farber Cancer Institute, Boston, MA

Past Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

VICE CHAIRS

North America Paul Carpenter, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Europe Katarina Le Blanc, MD, PhD, Karolinska University Hospital, Centre for Allogeneic Stem Cell Transplantation, Stockholm, Sweden

Central / South America Nelson Hamerschlak, MD, PhD, Albert Einstein Hospital, Sao Paolo, Brazil

Asia / Africa / Australia Biju George, MBBS, MD, Christian Medical College Hospital, Vellore, India

APPOINTED MEMBERS ASBMT Representative John DiPersio, MD, PhD, Washington University School of Medicine, St. Louis,

MO

Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA

Business Representative Jeff Chell, MD, NMDP/Be The Match, Minneapolis, MN

Collection Center Representative James Mason, MD, Scripps Blood and Marrow Transplant Program, San Antonio, TX

Cord Blood Bank Representative Andromachi Scaradavou, MD, New York Blood Center, Long Island City, NY

Donor Center Representative TBD

Patient / Family Representatives Jeffrey Haertling, Tierra Verde, FL

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CIBMTR 2018 Annual Report (Co-Chairs, Consumer Advocacy Committee)

EX OFFICIO MEMBERS

Hillary Hall, Cambridge, MA

Executive Director Randy Mills, PhD, NMDP/Be The Match, Minneapolis, MN

Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI

APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP

Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI

Senior Scientific Director SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI

Associate Scientific Director CIBMTR Minneapolis Steven Devine, MD, CIBMTR, Minneapolis, MN

Vice President CIBMTR Minneapolis Roberta King, MPH, CIBMTR, Minneapolis, MN

Senior Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI

Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN

Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN

NMDP/Be the Match / MCW / HRSA Contracting Officer Representative Nawraz Shawir, MBBS

NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret)

MCW / HRSA Contracting Officer Representatives Janet Kuramoto-Crawford, PhD, MHS Marilyn Levi, MD

MCW / NCI Project Officer William Merritt, PhD, Bethesda, MD MCW / NHLBI Project Officers Nancy DiFronzo, PhD, Bethesda, MD

Nahed El Kassar, MD, PhD, Bethesda, MD

MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD

Nominating Committee Chair Corey Cutler, MD, MPH, Dana Farber Cancer Institute, Boston MA

APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP

APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP

The Consumer Advocacy Committee communicates research results and data to the non-medical community and provides patient and donor perspectives during the development of the CIBMTR research agenda. Many committee members have personal experience as a donor, recipient, or family member of a recipient.

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CIBMTR 2018 Annual Report CHAIRS

Jeffery Haertling, Tierra Verde, FL Hilary

Hall, Cambridge, MA

MEMBERS

Nicole Adams, San Angelo, TX

Jack Aiello, MS, San Jose, CA

Maureen Beaman, MBA, Milwaukee, WI

James Omel, MD, (retired professional) Grand Island, NE

Kristin Scheeler, Leukemia and Lymphoma Society, White Plains, NY

Bryce Waldman, Chicago, IL

Jennifer Wilder, National Institutes of Health, Bethesda, MD

SCIENTIFIC DIRECTOR

J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI

EX OFFICIO MEMBERS

Linda Burns, MD, NMDP/ Be the Match and CIBMTR, Minneapolis, MN

Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN

Carol Doleysh, (CIBMTR liaison) CIBMTR, Milwaukee, WI

Mary Horowitz, MD, MS, CIBMTR, Milwaukee, WI

Janet Kuramoto-Crawford, PhD, MHS, Health Resources and Services Administration, Rockville, MD

Marilyn Levi, MD, Health Resources and Services Administration, Rockville, MD

Jennifer Motl, CIBMTR, Milwaukee, WI

Elizabeth Murphy, EdD, (NMDP/Be The Match liaison) NMDP/Be The Match, Minneapolis, MN

Bronwen Shaw, MD, PhD, CIBMTR, Milwaukee, WI

Nawraz Shawir, MBBS, Health Resources and Services Administration, Rockville, MD

Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP

APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP

The Nominating Committee consists of five members elected by the CIBMTR Assembly. Following an annual call for nominations, the Nominating Committee prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees. Elections are held

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CIBMTR 2018 Annual Report annually by confidential electronic ballot. The Nominating Committee also makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments after seeking recommendations from the CIBMTR Assembly, Advisory Committee, and incumbent Working Committee Chairs. All terms begin on March 1.

CHAIR

Corey Cutler, MD, MPH, Dana Farber Cancer Institute, Boston MA

MEMBERS

Ephraim Fuchs, MD, Johns Hopkins University Sidney Kimmel Cancer Center, Baltimore, MD

Mitchell Horwitz, MD, Duke University Medical Center, Durham, NC

Richard Maziarz, MD, Oregon Health and Science University, Portland, OR

Steven Pavletic, MD, MS, NIH - NCI Experimental Transplantation and Immunology Branch, Bethesda, MD

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CIBMTR 2018 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP

APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP

For information on Scientific Working Committee structure and organization, see Section 1.3.1. For information on Working Committee studies and their accomplishments, see Section 2.1.1.

ACUTE LEUKEMIA WORKING COMMITTEE

Chairs Marcos de Lima, MD, University Hospitals Case Medical Center Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Mark Litzow, MD, Mayo Clinic Rochester

Scientific Director Daniel Weisdorf, MD, CIBMTR Asst Scientific Dir Wael Saber, MD, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Jonathan Sanchez-Garcia, MPH, CIBMTR

AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE

Chairs Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute Peiman Hematti, MD, University of Wisconsin Hospital and Clinics

Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Rep Hillary Hall Statistician Kyle Bo-Subait, MPH, CIBMTR

CHRONIC LEUKEMIA WORKING COMMITTEE

Chairs Uday Popat, MD, MD Anderson Cancer Center Ronald Sobecks, MD, Cleveland Clinic Foundation Bart Scott, MD, Fred Hutchinson Cancer Research Center

Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Directors Ying Liu, PhD, CIBMTR Statistician Noel Estrada-Merly, MPH, CIBMTR

DONOR HEALTH AND SAFETY WORKING COMMITTEE

Chairs Galen Switzer, PhD, University of Pittsburgh Medical Center Michael Pulsipher, MD, Children’s Hospital of Los Angeles

Nirali Shah, MD, MHSc, National Cancer Institute – NIH Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Ex Officio Sr Advisor Dennis Confer, MD, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Consumer Adv Reps Jeffrey Haertling

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Hillary Hall Statisticians Jennifer Sees, MPH, CIBMTR

GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE

Chairs Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside Hospital

Ian McNiece, PhD, MD Anderson Cancer Center Claudio Brunstein, MD, PhD, University of Minnesota

Scientific Director Mary Eapen, MD, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Mariam Johnson, MPH, CIBMTR

GRAFT-VERSUS-HOST DISEASE WORKING COMMITTEE

Chairs Amin Alousi, MD, MD Anderson Cancer Center Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute Madan Jagasia, MBBS, MS, Vanderbilt University Medical Center

Scientific Directors Mukta Arora, MD, MS, CIBMTR Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Ying Liu, PhD, CIBMTR Consumer Adv Reps Kristin Scheeler, Leukemia and Lymphoma Society Jennifer Wilder, BSN, RN, National Institutes of Health Statistician Michael Hemmer, MS, CIBMTR

HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE

Chairs Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital William Wood, MD, MPH, University of North Carolina Hospitals Shahrukh Hashmi, MD, MPH, King Faisal Specialist Hospital and Research Center

Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Rep Jack Aiello Statistician Naya He, MPH, CIBMTR

IMMUNOBIOLOGY WORKING COMMITTEE

Chairs Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center Sophie Paczesny, MD, PhD, Indiana University Hospital/Riley Hospital for Children

Scientific Directors Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR

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Statistician Michelle Kuxhausen, MS, CIBMTR

INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE

Chairs Caroline Lindemans, MD, PhD, University Medical Center Utrecht Krishna Komanduri, MD, University of Miami Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center

Scientific Director Marcie Riches, MD, MS, CIBMTR, University of North Carolina Hospitals Statistical Directors Soyoung Kim, PhD, CIBMTR Statistician Min Chen, MS, CIBMTR

LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE

Chairs Mary Flowers, MD, Fred Hutchinson Cancer Research Center Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute - NIH David Buchbinder, MD, Children’s Hospital of Orange County

Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Asst Scientific Dir Rachel Phelan, MD, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Consumer Adv Reps Hillary Hall Jim Omel Statistician Stephanie Bo-Subait, MPH, CIBMTR

LYMPHOMA WORKING COMMITTEE

Chairs Anna Sureda, MD, PhD, Institut Catala d'Oncologia Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital Mohamed Kharfan-Dabaja, MD, MBA, Mayo Clinic Florida

Scientific Director Mehdi Hamadani, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician

Carlos Litovich, MPH, CIBMTR

PEDIATRIC CANCER WORKING COMMITTEE

Chairs Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center Angela Smith, MD, MS, University of Minnesota Medical Center Gregory Yanik, MD, The University of Michigan

Scientific Director Mary Eapen, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician TBD

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PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE

Chairs Tomer Mark, MD, University of Colorado Hospital Shaji Kumar, MD, Mayo Clinic Rochester Nina Shah, MD, University of California San Francisco Medical Center

Scientific Director Parameswaran Hari, MD, MS, CIBMTR Asst Scientific Dir Anita D’Souza, MD, CIBMTR Statistical Directors Raphael Fraser, PhD, CIBMTR Statistician Omar Davila, MPH, CIBMTR

PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND OTHER NON-MALIGNANT MARROW DISORDERS WORKING COMMITTEE

Chairs Jaap-Jan Boelens, MD, PhD, University Medical Center Utrecht Vikram Mathews, MD, Christian Medical College Hospital Christopher Dvorak, MD

Scientific Director Mary Eapen, MD, CIBMTR Statistical Director Soyoung Kim, PhD, CIBMTR Statistician Kyle Hebert, MS

REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE

Chairs Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania Medical Center Shin Mineishi, MD, Penn State Hershey Medical Center Edward Stadtmauer, MD, Abramson Cancer Center University of Pennsylvania

Medical Center Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statistician Caitrin Fretham, MPH, CIBMTR

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CIBMTR 2018 Annual Report APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP

APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP

The NMDP/Be The Match Histocompatibility Advisory Group also serves as the CIBMTR Immunobiology Steering Committee. This committee reviews and approves the use of donorrecipient specimens from the Research Repository in CIBMTR studies.

CHAIR

Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center, Tampa, FL

ADVISORY GROUP MEMBERS

Robyn Ashton, RN, MSN, HRSA Division of Transplantation, Rockville, MD

Juliet Barker, MD, Memorial Sloan Kettering Cancer Center, New York, NY Mary

Eapen, MD, MS, Medical College of Wisconsin, Milwaukee, WI

Marcelo Fernandez-Viña, PhD, Stanford Hospital and Clinics, Palo Alto, CA

Robert Hartzman, MD, Capt. MC, USN (Ret.), Navy Representative, C.W. Bill Young Marrow Donor Recruitment and Research Program, Rockville, MD

Carolyn K. Hurley, PhD, Diplomate ABHI, Georgetown University Hospital, Washington DC

Brent Logan, PhD, Medical College of Wisconsin, Milwaukee, WI

Carlheinz Mueller, MD, PhD, German National Bone Marrow Donor Registry (ZKRD), Ulm, Germany

Miguel Angel Perales, MD, Memorial Sloan Kettering Cancer Center Raja Rajalingham, PhD, University of California, San Francisco, CA

Bronwen Shaw, MD, PhD, Medical College of Wisconsin, Milwaukee, WI

NATIONAL MARROW DONOR PROGRAM (NMDP) STAFF

Dennis Confer, MD, CIBMTR

Steven Devine, MD, CIBMTR

Jason Dehn, MPH, NMDP

Martin Maiers, MS, CIBMTR

Stephen Spellman, MBS, CIBMTR

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CIBMTR 2018 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP

APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP

The Clinical Trials Advisory Committee make recommendations regarding the RCI BMT’s strategy, direction, and alignment with the CIBMTR’s scientific agenda (Section 2.3.2).

CHAIR

John Koreth, MBBS, DPhil, Dana Farber Cancer Institute, Boston, MA

MEMBERS

Andrea Artz, MD, MS, University of Chicago Medicine, Chicago, IL

Natalie Callander, MD, University of Wisconsin Hospital and Clinics, Madison, WI

Stephan Grupp, MD, PhD, Children’s Hospital of Philadelphia, Philadelphia, PA

Amrita Krishnan, MD, City of Hope, Duarte, CA

Shaji Kumar, MD, Mayo Clinic Rochester, Rochester, MN

Marco Mielcarek, MD, Fred Hutchinson Cancer Research Center, Seattle, WA

Bipin Savani, MD, Vanderbilt University Medical Center, Nashville, TN

APPOINTED MEMBERS

Jeffrey Haertling, Tierra Verde, FL Hilary

Hall, Cambridge, MA

EX OFFICIO MEMBERS

Linda Burns, MD, CIBMTR, Minneapolis, MN

Julie Clark, JD, NMDP/Be The Match, Minneapolis, MN

Dennis Confer, MD, CIBMTR, Minneapolis, MN

Steven Devine, MD, CIBMTR, Minneapolis, MN

Robert Hartzman, MD, Department of Defense, Rockville, MD

Mary Horowitz, MD, MS, CIBMTR, Milwaukee, WI

Roberta King, CIBMTR, Minneapolis, MN

Erin Leckrone, CIBMTR, Minneapolis, MN

Brent Logan, PhD, CIBMTR, Milwaukee, WI

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CIBMTR 2018 Annual Report Marcie Riches, MD, MS, University of North Carolina Hospitals, Chapel Hill, NC

Bronwen Shaw, MD, PhD, CIBMTR, Milwaukee, WI

Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN

APPENDIX D: PUBLICATIONS

The PMCID number is assigned by PubMed Central, the NIH’s free digital archive of biomedical and life sciences journal literature, and is in compliance with the NIH policy on public access.

APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

The following publications were generated by Scientific Working Committees within the Clinical Outcomes Research Program. For more information about the Working Committees, see Section 2.1.1.

SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Buchbinder D, Kelly DL, Duarte RF, Auletta JJ, Bhatt N, Byrne M, DeFilipp Z, Gabriel M, Mahindra A, Norkin M, Schoemans H, Shah AJ, Ahmed I, Atsuta Y, Basak GW, Beattie S, Bhella S, Bredeson C, Bunin N, Dalal J, Daly A, Gajewski J, Gale RP, Galvin J, Hamadani M, Hayashi RJ, Adekola K, Law J, Lee CJ, Liesveld J, Malone AK, Nagler A, Naik S, Nishihori T, Parsons SK, Scherwath A, Schofield HL, Soiffer R, Szer J, Twist I, Warwick AB, Wirk BM, Yi J, Battiwalla M, Flowers MDE, Savani B, Shaw BE

Neurocognitive dysfunction in hematopoietic cell transplant recipients: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Complications and Quality of Life Working Party of the EBMT

Bone Marrow Transplantation. 2018 May 1; 53(5):535-555. doi:10.1038/s41409-0170055-7. Epub 2018 Jan 17

PMC5985976

Lee C, Haneuse S, Wang H-L, Rose S, Spellman SR, Verneris MR, Hsu KC, Fleischhauer K, Lee SJ, Abdi R

Prediction of acute graftversus-host disease following hematopoietic cell transplantation

PLoS One. 13(1):e0190610. doi:10.1371/journal.pone.01 90610. Epub 2018 Jan 18

PMC5773230

Drobyski WR, Szabo A, Zhu F, Keever-Taylor C, Hebert KM, Dunn R, Yim S, Johnson B, D'Souza A, Eapen M, Fenske TS, Hari P, Hamadani M, Horowitz MM, Rizzo JD, Saber W, Shah N, Shaw B,

Tocilizumab, tacrolimus and methotrexate for the prevention of acute graftversus-host disease: Low incidence of lower gastrointestinal tract disease

Haematologica. 2018 Apr 1; 103(4):717-727. doi:10.3324/haematol.2017. 183434. Epub 2018 Jan 19

PMC5865423

Page | 138

CIBMTR 2018 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Pasquini M

Page | 139

CIBMTR 2018 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

SCIENTIFIC WORKING COMMITTEE PUBLICATIONS

Authors Title Citation PMCID Turcotte LM, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel D, Alousi A, Pidala J, Abdel-Azim H, Ahmed I, Beitinjaneh A, Buchbinder D, Byrne M, Callander N, Chao N, Choi SW, DeFilipp Z, Gadalla SM, Gale RP, Gergis U, Hashmi S, Hematti P, Holmberg L, Inamoto Y, Kamble RT, Lehmann L, MacMillan MA, McIver Z, Nishihori T, Norkin M, O'Brien T, Olsson RF, Reshef R, Saad A, Savani BN, Schouten HC, Seo S, Solh M, Verdonck L, Vij R, Wirk B, Yared J, Horowitz MM, Knight JM, Verneris MR

Donor body mass index does not predict graft versus host disease following hematopoietic cell transplantation

Bone Marrow Transplantation. 2018 Jul 1; 53(7):932-937. doi:10.1038/ s41409-018-0100-1. Epub 2018 Jan 30

PMC6041147

Buturovic L, Shelton J, Spellman SR, Wang T, Friedman L, Loftus D, Hesterberg L, Woodring T, Fleischhauer K, Hsu KC, Verneris MR, Haagenson M, Lee SJ

Evaluation of a machine learning-based prognostic model for unrelated hematopoietic cell transplantation donor selection

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jun 1; 24(6):1299-1306. doi:10.1016/j.bbmt.2018.01. 038. Epub 2018 Feb 1

PMC5993610

McCurdy SR, Zhang M-J, St Martin A, Al Malki MM, Bashey A, Gaballa S, Keesler DA, Hamadani M, Norkin M, Perales M-A, Reshef R, Rocha V, Romee R, Solh M, Urbano-Ispizua A, Waller EK, Fuchs EJ, Eapen M

Effect of donor characteristics on haploidentical transplantation with posttransplantation cyclophosphamide

Blood Advances. 2018 Feb 13; 2(3):299-307. doi:10.1182/bloodadvances. 2017014829. Epub 2018 Feb 9

PMC5812334

Sureda A, Zhang M-J, Dreger P, Carreras J, Fenske T, Finel H, Schouten H, Montoto S, Robinson S, Smith SM, Boumedil A, Hamadani M, Pasquini MC

Allogeneic hematopoietic stem cell transplantation for relapsed follicular lymphoma: A combined analysis on behalf of the Lymphoma Working Party of the EBMT and the Lymphoma Committee of the CIBMTR

Cancer. 2018 Apr 15; 124(8):1733-1742. doi:10.1002/cncr.31264. Epub 2018 Feb 9

PMC5946312

Shaw BE, Logan BR, Spellman SR, Marsh SGE, Robinson J, Pidala J, Hurley C, Barker J, Maiers M, Dehn J, Wang H, Haagenson M, Porter D, Petersdorf EW, Woolfrey A, Horowitz MM, Verneris M, Hsu KC, Fleischhauer K, Lee SJ

Development of an unrelated donor selection score predictive of survival after HCT: Donor age matters most

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 May 1; 24(5):1049-1056. doi:10.1016/j.bbmt.2018.02. 006. Epub 2018 Feb 14

PMC5953795

Page | 140


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Stiff PJ, Montesinos P, Peled T, Landau E, Rosenheimer Goudsmid NR, Mandel J, Hasson N, Olesinski E, Glukhman E, Snyder DA, Galamidi Cohen EG, Srur Kidron OS, Bracha D, Harati D, Ben-Abu K, Freind E, Freedman LS, Cohen YC, Olmer L, Barishev R, Rocha V, Gluckman E, Horowitz MM, Eapen M, Nagler A, Sanz G

Cohort-controlled comparison of umbilical cord blood transplantation using carlecortemcel-L, a single progenitor-enriched cord blood, to double cord blood unit transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jul 1; 24(7):1463-1470. doi:10.1016/j.bbmt.2018.02. 012. Epub 2018 Mar 1

PMC6045964

D'Souza A, Millard H, Knight J, Brazauskas R, Lee SJ, Flynn KE, Rizzo JD, Shaw BE

Prevalence of self-reported sleep dysfunction before allogeneic hematopoietic cell transplantation

Bone Marrow Transplantation. 2018 Aug 1; 53(8):1079-1082. doi:10.1038/s41409-0180150-4. Epub 2018 Mar 7

PMC6098949

Wood WA, Brazauskas R, Hu ZH, Abdel-Azim H, Ahmed IA, Aljurf M, Badawy S, Beitinjaneh A, George B, Buchbinder D, Cerny J, Dedeken L, Diaz MA, Freytes CO, Ganguly S, Gergis U, Almaguer DG, Gupta A, Hale G, Hashmi SK, Inamoto Y, Kamble RT, Adekola K, Kindwall-Keller T, Knight J, Kumar L, Kuwatsuka Y, Law J, Lazarus HM, LeMaistre C, Olsson RF, Pulsipher MA, Savani BN, Schultz KR, Saad AA, Seftel M, Seo S, Shea TC, Steinberg A, Sullivan K, Szwajcer D, Wirk B, Yared J, Yong A, Dalal J, Hahn T, Khera N, Bonfim C, Atsuta Y, Saber W

Country-level macroeconomic indicators predict early postallogeneic hematopoietic cell transplantation survival in acute lymphoblastic leukemia: A CIBMTR analysis

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Sep 1; 24(9):1928-1935. doi:10.1016/j.bbmt.2018.03. 016. Epub 2018 Mar 19

PMC6146070

Switzer GE, Macis M, Fabi R, Abress L, Confer D, Bruce J, Howe K, Fowler S, McNulty M, Pastorek G, Dew MA

Providing level-of-match information to perfectly matched unrelated donors: Evaluating acceptability and potential changes in donor availability

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.03. 017. Epub 2018 Mar 21

N/A

Duncan CN, Brazauskas R, Huang J, Shaw BE, Majhail NS, Savani BN, Flowers MED, Battiwalla M, Beebe K, Dietz AC, Dvorak CC, Giller R, Jacobsohn DA, Kletzel M, Martin PL, Nemecek ER, Nuechterlein B, Talano J-A, Pulsipher MA, Baker KS

Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation

Bone Marrow Transplantation. 2018 Oct 1; 53(10):1278-1287. doi:10.1038/s41409-0180155-z. Epub 2018 Mar 26

PMC6158112

Page | 141


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Zhu Q, Yan L, Liu Q, Zhang C, Wei L, Hu Q, Preus L, Clay-Gilmour AI, Onel K, Stram DO, Pooler L, Sheng X, Haiman CA, Zhu X, Spellman SR, Pasquini M, McCarthy PL, Liu S, Hahn T, Sucheston-Campbell LE

Exome chip analyses identify genes affecting mortality after HLA-matched unrelated-donor blood and marrow transplantation

Blood. 2018 May 31; 131(22):2490-2499. doi:10.1182/blood-2017-11817973. Epub 2018 Apr 2

PMC5981168

Gadalla SM, Aubert G, Wang T, Haagenson M, Spellman SR, Wang L, Katki HA, Savage SA, Lee SJ

Donor telomere length and causes of death after unrelated hematopoietic cell transplantation in patients with marrow failure

Blood. 2018 May 31; 131(21):2393-2398. doi:10.1182/blood-2017-10812735. Epub 2018 Apr 9

PMC5969378

Smith SM, Godfrey J, Ahn KW, DiGilio A, Ahmed S, Agrawal V, Bachanova V, Bacher U, Bashey A, Bolaños-Meade J, Cairo M, Chen A, Chhabra S, Copelan E, Dahi PB, Aljurf M, Farooq U, Ganguly S, Hertzberg M, Holmberg L, Inwards D, Kanate AS, Karmali R, Kenkre VP, Kharfan-Dabaja MA, Klein A, Lazarus HM, Mei M, Mussetti A, Nishihori T, Ramakrishnan Geethakumari P, Saad A, Savani BN, Schouten HC, Shah N, Urbano-Ispizua A, Vij R, Vose J, Sureda A, Hamadani M

Autologous transplantation versus allogeneic transplantation in patients with follicular lymphoma experiencing early treatment failure

Cancer. 2018 Jun 15; 124(12):2541-2551. doi:10.1002/cncr.31374. Epub 2018 Apr 12

PMC5990449

Horowitz M, Schreiber H, Elder A, Heidenreich O, Vormoor J, Toffalori C, Vago L, Kröger N

Epidemiology and biology of relapse after stem cell transplantation

Bone Marrow Transplantation. 2018 Nov 1; 53(11):1379-1389. doi:10.1038/s41409-0180171-z. Epub 2018 Apr 18

PMC6282701

Shah NN, Ahn KW, Litovich C, Fenske TS, Ahmed S, Battiwalla M, Bejanyan N, Dahi PB, BolañosMeade J, Chen AI, Ciurea SO, Bachanova V, DeFilipp Z, Epperla N, Farhadfar N, Herrera AF, Haverkos BM, Holmberg L, Hossain NM, Kharfan-Dabaja MA, Kenkre VP, Lazarus HM, Murthy HS, Nishihori T, Rezvani AR, D'Souza A, Savani BN, Ulrickson ML, Waller EK, Sureda A, Smith SM, Hamadani M

Outcomes of Medicare-age eligible NHL patients receiving RIC allogeneic transplantation: A CIBMTR analysis

Blood Advances. 2018 Apr 24; 2(8):933-940. doi:10.1182/bloodadvances. 2018018531. Epub 2018 Apr 23

PMC5916010

Authors Title Citation PMCID

Page | 142


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai S-Y

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

Blood. 2018 Jun 28; 131(26):2967-2977. doi:10.1182/blood-2017-10809822. Epub 2018 May 4

PMC6024640

Kumar AJ, Kim S, Hemmer MT, Arora M, Spellman SR, Pidala JA, Couriel DR, Alousi AM, Aljurf MD, Cahn J-Y, Cairo MS, Cutler CS, Farhan S, Gergis U, Hale GA, Hashmi SK, Inamoto Y, Kamble RT, Kharfan-Dabaja MA, MacMillan ML, Marks DI, Nakasone H, Norkin M, Qayed M, Ringden O, Schouten HC, Schultz KR, Solh MM, Teshima T, Urbano-Ispizua A, Verdonck LF, Gale RP, Hamilton BK, Majhail NS, Loren AW

Graft-versus-host disease in recipients of male unrelated donor compared with parous female sibling donor transplants

Blood Advances. 2018 May 8; 2(9):1022-1031. doi:10.1182/bloodadvances. 2017013052. Epub 2018 May 8

PMC5941995

Elgarten CW, Arnold SD, Li Y, Huang Y-V, Riches ML, Gerber JS, Aplenc R, Saber W, Fisher BT

Hospital-level variability in broad-spectrum antibiotic use for children with acute leukemia undergoing hematopoietic cell transplantation

Infection Control and Hospital Epidemiology. 2018 Jul 1; 39(7):797-805. doi:10.1016/j.spl.2017.11.014. Epub 2018 May 8

PMC6081961

Robinson TM, Fuchs EJ, Zhang M-J, St Martin A, Labopin M, Keesler DA, Blaise D, Bashey A, Bourhis J-H, Ciceri F, Ciurea SO, Devine SM, Mohty M, McCurdy SR, Milpied N, McNiece IK, Rocha V, Romee R, Socie G, Yakoub-Agha I, Soiffer RJ, Eapen M, Nagler A

Related donor transplants: Has posttransplantation cyclophosphamide nullified the detrimental effect of HLA mismatch?

Blood Advances. 2018 Jun 12; 2(11):1180-1186. doi:10.1182/bloodadvances. 2018018291. Epub 2018 May 24

PMC5998932

Fleischhauer K, Hsu KC, Shaw BE Prevention of relapse after allogeneic hematopoietic cell transplantation by donor and cell source selection

Bone Marrow Transplantation. doi:10.1038/s41409-018-0218-1. Epub 2018 May 24

N/A

Harris AC, Boelens JJ, Ahn KW, Fei M, Abraham A, Artz A, Dvorak C, Frangoul H, Freytes C, Gale RP, Hong S, Lazarus HM, Loren A, Mineishi S, Nishihori T, O'Brien T, Williams K, Pasquini MC, Levine JE

Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine

Blood Advances. 2018 Jun 12; 2(11):1198-1206. doi:10.1182/bloodadvances. 2018016956. Epub 2018 May 29

PMC5998928


Page | 143


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Lee SJ, Onstad L, Chow EJ, Shaw BE, Jim HSL, Syrjala KL, Baker KS, Buckley S, Flowers ME

Patient-reported outcomes and health status associated with chronic graft-versus-host disease

Haematologica. 2018 Sep 1; 103(9):1535-1541. doi:10.3324/haematol.2018. 192930. Epub 2018 Jun 1

PMC6119141

Wang Y, Zhou W, Alter BP, Wang T, Spellman SR, Haagenson M, Yeager M, Lee SJ, Chanock SJ, Savage SA, Gadalla SM

Chromosomal aberrations and survival after unrelated donor hematopoietic stem cell transplant in patients with Fanconi anemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Oct 1; 24(10):2003-2008. doi:10.1016/j.bbmt.2018.05. 027. Epub 2018 Jun 4

PMC6239962

Schoemans HM, Lee SJ, Ferrara JL, Wolff D, Levine JE, Schultz KR, Shaw BE, Flowers ME, Ruutu T, Greinix H, Holler E, Basak G, Duarte RF, Pavletic SZ

EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-versus-host disease assessment

Bone Marrow Transplantation. doi:10.1038/s41409-0180204-7. Epub 2018 Jun 5

N/A

Petersdorf EW, Stevenson P, Malkki M, Strong RK, Spellman SR, Haagenson MD, Horowitz MM, Gooley T, Wang T

Patient HLA germline variation and transplant survivorship

Journal of Clinical Oncology. 2018 Aug 20; 36(24):25242531. doi:10.1200/JCO.2017.77.6534. Epub 2018 Jun 14

PMC6097831

Bhatt NS, Brazauskas R, Tecca HR, Vogel J, Mattila D, Lee SJ, Horowitz MM, Rizzo JD, Shaw BE

Female sex is associated with poor health-related quality of life in children at 12 months post-hematopoietic cell transplantation

Journal of Pediatric Hematology/Oncology. doi:10.1097/MPH.00000000 00001239. Epub 2018 Jun 19

PMC6301126

Strouse C, Zhang Y, Zhang M-J, DiGilio A, Pasquini M, Horowitz MM, Lee S, Ho V, Ramanathan M, Chinratanalab W, Loren A, Burns LJ, Artz A, Villa KF, Saber W

Risk score for the development of veno-occlusive disease after allogeneic hematopoietic cell transplant


PMC6239945

Sullivan KM, Majhail NS, Bredeson C, Carpenter PA, Chatterjee S, Crofford LJ, Georges GE, Nash RA, Pasquini MC, Sarantopoulos S, Storek J, Savani B, St. Clair EW

Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: Position statement from the American Society for Blood and Marrow Transplantation


PMC6239926

Authors Title Citation PMCID Sarhan M, Brandt L, Felices M, Guldevall K, Lenvik T, Hinderlie P, Curtsinger J, Warlick E, Spellman SR,

161533 TriKE stimulates NK-cell function to overcome myeloidderived suppressor cells

Blood Advances. doi:10.1182/bloodadvances. 2017012369. Epub 2018 Jun

PMC6020813

Page | 144


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Blazar BR, Weisdorf D, Cooley S, Vallera DA, Önfelt B

in MDS

25

Radivoyevitch T, Dean RM, Shaw BE, Brazauskas R, Tecca HR, Molenaar RJ, Battiwalla M, Savani BN, Flowers MED, Cooke KR, Hamilton BK, Kalaycio M, Maciejewski JP, Ahmed I, Akpek G, Bajel A, Buchbinder D, Cahn J-Y, D'Souza A, Daly A, DeFilipp Z, Ganguly S, Hamadani M, Hayashi RJ, Hematti P, Inamoto Y, Khera N, Kindwall-Keller T, Landau H, Lazarus H, Majhail NS, Marks DI, Olsson RF, Seo S, Steinberg A, William BM, Wirk B, Yared JA, Aljurf M, Abidi MH, Allewelt H, Beitinjaneh A, Cook R, Cornell RF, Fay JW, Hale G, Chakrabarty JH, Jodele S, Kasow KA, Mahindra A, Malone AK, Popat U, Rizzo JD, Schouten HC, Warwick AB, Wood WA, Sekeres MA, Litzow MR, Gale RP, Hashmi SK

Risk of acute myeloid leukemia and myelodysplastic syndrome after autotransplants for lymphomas and plasma cell myeloma

Leukemia Research. 2018 Nov 1; 74:130-136. doi:10.1016/j.leukres.2018.07.016. Epub 2018 Jul 19

PMC6219911

Arrieta-Bolaños E, Crivello P, Shaw BE, Ahn KW, Wang H-L, Verneris MR, Hsu KC, Pidala J, Lee SJ, Fleischhauer K, Spellman SR

In silico prediction of nonpermissive HLA-DPB1 mismatches in unrelated HCT by functional distance

Blood Advances. 2018 Jul 24; 2(14):1773-1783. doi:10.1182/bloodadvances.2018019620. Epub 2018 Jul 24

PMC6058232

Eapen M Is a matched sibling the ideal donor for hematopoietic cell transplant?

Haematologica. 2018 Aug 1; 103(8):1251-1252. doi:10.3324/haematol.2018. 196980. Epub 2018 Aug 1

N/A

Nikiforow S, Wang T, Hemmer M, Spellman S, Akpek G, Antin JH, Choi SW, Inamoto Y, Khoury HJ, MacMillan M, Marks DI, Meehan K, Nakasone H, Nishihori T, Olsson R, Paczesny S, Przepiorka D, Reddy V, Reshef R, Schoemans H, Waller N, Weisdorf D, Wirk B, Horowitz M, Alousi A, Couriel D, Pidala J, Arora M, Cutler C

Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value in isolation or with other graftversus-host disease symptoms as currently diagnosed and treated

Haematologica. doi:10.3324/haematol.2017.182550. Epub 2018 Aug 3. 30076185

PMC6165812


Page | 145


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Liang J, Lee SJ, Storer BE, Shaw BE, Chow EJ, Flowers ME, Krakow EF, Bar M, Syrjala KL, Salit RB, Kurukulasuriya CE, Jim HSL

Rates and risk factors for posttraumatic stress disorder symptomatology among adult hematopoietic cell transplant recipients and their informal caregivers

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):145-150. doi:10.1016/ j.bbmt.2018.08.002. Epub2018 Aug 8

PMC6310630

Keesler DA, St. Martin A, Bonfim C, Seber A, Zhang M-J, Eapen M

Bone marrow versus peripheral blood from unrelated donors for children and adolescents with acute leukemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Dec 1; 24(12):2487-2492. doi:10.1016/j.bbmt.2018.08. 010. Epub 2018 Aug 21

PMC6286246

Eapen M, Brazauskas R, Hemmer M, Perez WS, Steinert P, Horowitz MM, Deeg HJ

Hematopoietic cell transplant for acute myeloid leukemia and myelodysplastic syndrome: Conditioning regimen intensity

Blood Advances. 2018 Aug 28; 2(16):2095-2103. doi:10.1182/bloodadvances. 2018021980. Epub 2018 Aug 22

PMC6113615

Turcotte LM, Wang T, Hemmer MT, Spellman SR, Arora M, Yingst A, Couriel D, Alousi A, Pidala J, Knight JM, Verneris MR

Pro-inflammatory cytokine and adipokine levels in adult unrelated marrow donors are not associated with hematopoietic cell transplantation outcomes

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):12-18. doi:10.1016/ j.bbmt.2018.08.011. Epub2018 Aug 22

[PMC journal - in process]

Scott EC, Hari P, Kumar S, Fraser R, Davila O, Shah N, Gale RP, Diaz MA, Agrawal V, Cornell RF, Ganguly S, Akpek G, Freytes C, Hashmi S, Malek E, Kamble RT, Lazarus H, Solh M, Usmani SZ, Kanate AS, Saad A, Chhabra S, Gergis U, Cerny J, Kyle RA, Lee C, Kindwall-Keller T, Assal A, Hildebrandt GC, Holmberg L, Maziarz RT, Nishihori T, Seo S, Kumar S, Mark T, D'Souza A

Staging systems for newly diagnosed myeloma patients undergoing autologous hematopoietic cell transplantation: The revised international staging system shows the most differentiation between groups

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Dec 1; 24(12):2443-2449. doi:10.1016/j.bbmt.2018.08. 013. Epub 2018 Aug 22

PMC6293469

Weisdorf D, Cooley S, Wang T, Trachtenberg E, Haagenson MD, Vierra-Green C, Spellman SR, Spahn A, Vogel J, Kobusingye H, Fehninger T, Woolfrey A, Devine S, Ross M, Waller EK, Sobecks R, Parham P, Guethlein LA, Marsh SGE, Miller J

KIR donor selection: feasibility in identifying better donors

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):e28-e32. doi:10.1016/ j.bbmt.2018.08.022. Epub2018 Aug 24

PMC6310641

Page | 146


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Chhabra S, Liu Y, Hemmer MT, Costa L, Pidala JA, Couriel DR, Alousi A, Majhail NS, Stuart RK, Kim D, Ringden O, Urbano-Ispizua A, Saad A, Savani BN, Cooper B, Marks DI, Socie G, Schouten HC, Schoemans H, Abdel-Azim H, Yared J, Cahn J-Y, Wagner J, Antin JH, Verdonck LF, Lehmann L, Aljurf MD, MacMillan ML, Litzow MR, Solh MM, Qayed M, Hematti P, Kamble RT, Vij R, Hayashi RJ, Gale RP, Martino R, Seo S, Hashmi SK, Nishihori T, Teshima T, Gergis U, Inamoto Y, Spellman SR, Arora M, Hamilton BK

Comparative analysis of calcineurin-inhibitor-based methotrexate and mycophenolate mofetilcontaining regimens for prevention of graft-versus-host disease after reduced intensity conditioning allogeneic transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):73-85. doi:10.1016/ j.bbmt.2018.08.018. Epub 2018 Aug 25


Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai S-Y, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Blood. 2018 Oct 25; 132(17):1737-1749. doi:10.1182/blood-2018-03840702. Epub 2018 Aug 28

PMC6202916

Marsh RA, Hebert KM, Keesler D, Boelens JJ, Dvorak CC, Eckrich MJ, Kapoor N, Parikh S, Eapen M

Practice pattern changes and improvements in hematopoietic cell transplantation for primary immunodeficiencies

Journal of Allergy and Clinical Immunology. 2018 Dec 1; 142(6):2004-2007. doi:10.1016/j.jaci.2018.08.010. Epub 2018 Aug 28

PMC6289686

Page | 147


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Jones RJ, Horowitz MM Should an HLA-matched donor

still be considered the perfect donor?

The Lancet Haematology. 5(9):e388-e390. doi:10.1016/S2352-3026(18)30119-4. Epub 2018 Sep 1

N/A

Authors Title Citation PMCID Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai S-Y, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM

The genetic landscape of SCID in the US and Canada in the current era (2010-2018)

Journal of Allergy and Clinical Immunology. doi:10.1016/j.jaci.2018.08.027. Epub 2018 Sep 5


Rice C, Eikema DJ, Marsh JCW, Knol C, Hebert KM, Putter H, Peterson E, Deeg HJ, Halkes CJM, Pidala J, Anderlini P, Tischer J, Kroger N, McDonald A, Antin JH, Schaap NPM, Hallek M, Einsele H, Mathews V, Kapoor N, Boelens JJ, Mufti GJ, Potter V, Pefault de la Tour R, Eapen M, Dufour C

Allogeneic hematopoietic cell transplantation in patients aged 50 years or older with severe aplastic anemia

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.08. 029. Epub 2018 Sep 5


Mangurian C, Scalchunes C, Yoo J, Logan B, Henderson T, Iyengar S, Smith H, Cowan MJ

Psychosocial services for primary immunodeficiency disorder families during hematopoietic cell transplantation: A descriptive study

Palliative & Supportive Care. doi:10.1017/S1478951518000603. Epub 2018 Sep 18

N/A

Woolfrey A, Wang T, Lee SJ, Haagenson MD, Chen G, Fleischhauer K, Horan J, Hsu K, Verneris M, Spellman SR, Fernandez-Vina M

Donor-specific anti-HLA antibodies in unrelated hematopoietic cell transplantation for nonmalignant disorders

Bone Marrow Transplantation. doi:10.1038/s41409-0180334-y. Epub 2018 Sep 19


Lund TC, Ahn KW, Tecca HR, Hilgers MV, Abdel-Azim H, Abraham A, Diaz MA, Badawy SM, Broglie L, Brown V, Dvorak CC, Gonzalez-Vicent M, Hashem H, Hayashi RJ, Jacobsohn DA, Kent MW, Li C-K, Margossian SP, Martin PL, Mehta P, Myers K, Olsson R, Page K, Pulsipher MA,

Outcomes after second hematopoietic cell transplant for children and young adults with relapsed acute leukemia



Page | 148


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Shaw PJ, Smith AR, Triplett BM, Verneris MR, Eapen M

Authors Title Citation PMCID Sahebi F, Garderet L, Kanate AS, Eikema DJ, Knelange NS, Alvelo OFD, Koc Y, Blaise D, Bashir Q, Moraleda JM, Dreger P, Sanchez JF, Ciurea S, Schouten HC, Shah NN, Verbeek M, Rösler W, Diez-Martin JL, Schoenland S, D'Souza A, Kröger N, Hari P

The outcome of haplo-identical transplantation in patients with relapsed multiple myeloma: An EBMT/CIBMTR report



Pingel J, Wang T, Hagenlocher Y, Hernández-Frederick CJ, Nagler A, Haagenson MD, Fleischhauer K, Hsu KC, Verneris MR, Lee SJ, Mohty M, Polge E, Spellman SR, Schmidt AH, van Rood JJ

The effect of NIMA matching in adult unrelated mismatched hematopoietic stem cell transplantation - a joint study of the Acute Leukemia Working Party of the EBMT and the CIBMTR

Bone Marrow Transplantation. doi:10.1038/s41409-018-0345-8. Epub 2018 Oct 2


Norkin M, Shaw BE, Brazauskas R, Tecca HR, Leather HL, Gea-Banacloche J, Kamble R, DeFilipp Z, Jacobsohn DA, Ringden O, Inamoto Y, Kasow K, Buchbinder D, Shaw P, Hematti P, Schears R, Badawy SM, Lazarus HM, Bhatt N, Horn B, Chhabra S, Page K, Hamilton B, Hildebrandt GC, Yared JA, Agrawal V, Beitinjaneh A, Majhail NS, Kindwall-Keller T, Olsson RF, Schoemans H, Gale RP, Ganguly S, Ahmed I, Schouten HC, Liesveld J, Khera N, Steinberg A, Shah AJ, Solh M, Marks DI, Rybka W, Aljurf M, Dietz AC, Gergis U, George B, Seo S, Flowers MED, Battiwalla M, Savani BN, Riches ML, Wingard JR

Characteristics of late fatal infections after allogeneic hematopoietic cell transplant

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.09. 031. Epub 2018 Oct 2


Alousi A, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel DR, Pidala J, Anderlini P, Boyiadzis M, Bredeson CN, Cahn J-Y, Cairo MS, Gadalla SM, Hashmi SK, Gale RP, Kanda J, Kamble RT, Kharfan-Dabaja MA, Litzow MR, Ringden O, Saad A, Schultz KR, Verdonck LF, Waller EK, Yared JA, Holtan SG, Weisdorf DJ

Peripheral blood versus bone marrow from unrelated donors: bone marrow allografts have improved longterm overall and graft-versushost disease, relapse-free survival

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.09. 004. Epub 2018 Oct 3



Page | 149


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Salman A, Koparde V, Hall CE, Jameson-Lee M, Roberts C, Serrano M, AbdulRazzaq B, Meier J, Kennedy C, Manjili MH, Spellman SR, Wijesinghe D, Hashmi S, Buck G, Qayyum R, Neale M, Reed J, Toor AA

Determining the quantitative principles of T cell response to antigenic disparity in stem cell transplantation

Frontiers in Immunology. doi:10.3389/fimmu.2018.02284. Epub 2018 Oct 11

PMC6193078

Misra MK, Augusto DG, Montero Martin G, Nemat-Gorgani N, Sauter J, Hofmann JA, Traherne JA, González-Quezada B, Gorodezky C, Bultitude W, Marin W, Vierra-Green C, Anderson KM, Balas A, CaroOleas JL, Cisneros E, Colucci F, Dandekar R, Elfishawi S, FernándezViña MA, Fouda M, GonzálezFernández R, Große A, HerreroMata MJ, Hollenbach SQ, Marsh SG, Mentzer A, Middleton D, Moffett A, Moreno-Hidalgo MA, Mossallam G, Nakimuli A, Oksenberg JR, Oppenheimer SJ, Parham P, Petzl-Erler ML, Planelles D, SánchezGarcía F, Sánchez-Gordo F, Schmidt AH, Trowsdale J, Vargas LB, Vicario JL, Vilches C, Norman PJ, Hollenbach JA

Report from the killer-cell immunoglobulin-like receptors (KIR) component of the 17th International HLA and Immunogenetics Workshop

Human Immunology. 2018 Dec 1; 79(12):825-833. doi:10.1016/j.humimm.2018 .10.003. Epub 2018 Oct 12


Bhatt NS, Brazauskas R, Tecca HR, Carreras J, Burns LJ, Phelan R, Salit RB, Syrjala KL, Talano JM, Shaw BE

Post-transplantation employment status of adult survivors of childhood allogeneic hematopoietic cell transplant: A report from the Center for International Blood and Marrow Transplant Research (CIBMTR)

Cancer. 2019 Jan 1; 125(1):144-152. doi:10.1002/cncr.31781. Epub 2018 Oct 12

PMC6310211

Wang Y, Savage SA, Alsaggaf R, Aubert G, Dagnall CL, Spellman SR, Lee SJ, Hicks B, Jones K, Katki HA, Gadalla SM

Telomere length calibration from qPCR measurement: Limitations of current method

Cells. 7(11):183. doi:10.3390/cells7110183. Epub 2018 Oct 24

PMC6262465


Page | 150


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Pulsipher MA, Logan BR, Kiefer DM, Chitphakdithai P, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Akard LP, Artz AS, Ball ED, Bayer R-L, Bigelow C, Bolwell BJ, Broun ER, Delgado DC, Duckworth K, Dvorak CC, Hahn TE, Haight AE, Hari PN, Hayes-Lattin BM, Jacobsohn DA, Jakubowski AA, Kasow KA, Lazarus HM, Liesveld JL, Linenberger M, Litzow MR, Longo W, MagalhaesSilverman M, McCarty JM, McGuirk JP, Mori S, Parameswaran V, Prasad VK, Rowley SD, Rybka WB, Sahdev I, Schriber JR, Selby GB, Shaughnessy PJ, Shenoy S, Spitzer T, Tse WT, Uberti JP, Vusirikala M, Waller EK, Weisdorf DJ, Yanik GA, Navarro WH, Horowitz MM, Switzer GE, Confer DL, Shaw BE

Related peripheral blood stem cell donors experience more severe symptoms and less complete recovery at 1-year compared to unrelated donors

Haematologica. doi:10.3324/haematol.2018. 200121. Epub 2018 Oct 31


Chhabra S, Ahn KW, Hu Z-H, Jain S, Assal A, Cerny J, Copelan EA, Daly A, DeFilipp Z, Gadalla SM, Gale RP, Ganguly S, Hamilton BK, Hildebrandt GC, Hsu JW, Inamoto Y, Kanate AS, Khoury HJ, Lazarus HM, Litzow MR, Nathan S, Olsson RF, Pawarode A, Ringden O, Rowe JM, Saad A, Savani BN, Schouten HC, Seo S, Shah NN, Solh M, Stuart RK, Ustun C, Woolfrey AE, Yared JA, Alyea EP, Kalaycio ME, Popat U, Sobecks R, Saber W

Myeloablative vs reducedintensity conditioning allogeneic hematopoietic cell transplantation for chronic myeloid leukemia

Blood Advances. 2018 Nov 13; 2(21):2922-2936. doi:10.1182/bloodadvances. 2018024844. Epub 2018 Nov 5

PMC6234373


Page | 151


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Pulsipher MA, Logan BR, Chitphakdithai P, Kiefer DM, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Varni JW, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Akard LP, Artz AS, Ball ED, Bayer R-L, Bigelow C, Bolwell BJ, Broun ER, Bunin NJ, Delgado DC, Duckworth K, Dvorak CC, Hahn TE, Haight AE, Hari PN, Hayes-Lattin BM, Jacobsohn DA, Jakubowski AA, Kasow KA, Lazarus HM, Liesveld JL, Linenberger M, Litzow MR, Longo W, MagalhaesSilverman M, McCarty JM, McGuirk JP, Mori S, Prasad VK, Rowley SD, Rybka WB, Sahdev I, Schriber JR, Selby GB, Shaughnessy PJ, Shenoy S, Spitzer T, Tse WT, Uberti JP, Vusirikala M, Waller EK, Weisdorf DJ, Yanik GA, Navarro WH, Horowitz MM, Switzer GE, Shaw BE, Confer DL

The effect of aging and predonation comorbidities on the related PBSC donor experience: A report from the Related Donor Safety Study (RDSafe)

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.11. 004. Epub 2018 Nov 10


Brunstein CG, Pasquini MC, Kim S, Fei M, Adekola K, Ahmed I, Aljurf M, Agrawal V, Auletta JJ, Battiwalla M, Bejanyan N, Bubalo J, Cerny J, Chee L, Ciurea S, Freytes C, Gadalla SM, Gale RP, Ganguly S, Hashmi SK, Hematti P, Hildebrandt G, Holmberg L, Lahoud OB, Landau H, Lazarus HM, de Lima M, Mathews V, Maziarz R, Nishihori T, Norkin M, Olsson R, Reshef R, Rotz S, Savani B, Schouten HC, Seo S, Wirk BM, Yared J, Mineishi S, Rogosheske J, Perales M-A

The effect of conditioning regimen dose reduction in obese patients undergoing autologous hematopoietic cell transplantation



Inamoto Y, Valdés-Sanz N, Ogawa Y, Alves M, Berchicci L, Galvin J, Greinix H, Hale GA, Horn B, Kelly D, Liu H, Rowley S, Schoemans H, Shah A, Stanghellini MTL, Argrawal V, Ahmed I, Ali A, Bhatt N, Byrne M, Chhabra S, DeFilipp Z, Fahnehjelm K, Farhadfar N, Horn E, Lee C, Nathan S, Penack O, Prasad P, Rotz S, Rovó A, Yared J, Pavletic S, Basak GW, Battiwalla M, Duarte R, Savani

Ocular graft-versus-host disease after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT



Page | 152


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS BN, Flowers ME, Shaw BE, Petriček I

Authors Title Citation PMCID Inamoto Y, Petriček I, Burns L, Chhabra S, DeFilipp Z, Hematti P, Rovó A, Schears R, Shah A, Argrawal V, Ahmed A, Ahmed I, Ali A, Aljurf M, Alkhateeb H, Beitinjaneh A, Bhatt N, Buchbinder D, Byrne M, Callander N, Fahnehjelm K, Farhadfar N, Gale RP, Ganguly S, Hildebrandt GC, Horn E, Jakubowski A, Kamble RT, Law J, Lee C, Nathan S, Penack O, Pingali R, Prasad P, Pulanic D, Rotz S, Shreenivas A, Steinberg A, Tabbara K, Tichelli A, Wirk B, Yared J, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers, Shaw BE, Valdés-Sanz N

Non-GVHD ocular complications after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.11. 033. Epub 2018 Dec 3


Inamoto Y, Valdés-Sanz N, Ogawa Y, Alves M, Berchicci L, Galvin J, Greinix H, Hale GA, Horn B, Kelly D, Liu H, Rowley S, Schoemans H, Shah A, Lupo Stanghellini MT, Argrawal V, Ahmed I, Ali A, Bhatt N, Byrne M, Chhabra S, DeFilipp Z, Fahnehjelm K, Farhadfar N, Horn E, Lee C, Nathan S, Penack O, Prasad P, Rotz S, Rovó A, Yared J, Pavletic S, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers MED, Shaw BE, Petriček I

Ocular graft-versus-host disease after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT

Bone Marrow Transplantation. doi:10.1038/s41409-0180340-0. Epub 2018 Dec 7


Inamoto Y, Petriček I, Burns L, Chhabra S, DeFilipp Z, Hematti P, Rovó A, Schears R, Shah A, Agrawal V, Ahmed A, Ahmed I, Ali A, Aljurf M, Alkhateeb H, Beitinjaneh A, Bhatt N, Buchbinder D, Byrne M, Callander N, Fahnehjelm K, Farhadfar N, Gale RP, Ganguly S, Hildebrandt GC, Horn E, Jakubowski A, Kamble RT, Law J, Lee C, Nathan S, Penack O, Pingali R, Prasad P, Pulanic D, Rotz S, Shreenivas A, Steinberg A, Tabbara K, Tichelli A, Wirk B, Yared J, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers MED, Shaw BE, Valdés-Sanz

Non-GVHD ocular complications after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT



Page | 153


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS N

Authors Title Citation PMCID Ustun C, Young J-H, Papanicolau GA, Kim S, Ahn KW, Chen M, AbdelAzim H, Aljurf M, Beitinjaneh A, Brown V, Cerny J, Chhabra S, Kharfan-Dabaja MA, Dahi PB, Daly A, Dandoy CE, Dvorak CC, Freytes CO, Hashmi S, Lazarus H, Ljungman P, Nishihori T, Page K, Pingali SRK, Saad A, Savani BN, Weisdorf D, Williams K, Wirk B, Auletta JJ, Lindemans CA, Komanduri K, Riches M

Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation



Thakar M, Broglie L, Logan B, Artz A, Bunin N, Burroughs LM, Fretham C, Jacobsohn DA, Loren AW, Kurtzberg J, Martinez CA, Mineishi S, Nelson AS, Woolfrey A, Pasquini MC, Sorror ML

The hematopoietic cell transplant comorbidity index predicts survival after allogeneic transplant for nonmalignant diseases

Blood. doi:10.1182/blood2018-09-876284. Epub 2018 Dec 13


Askar M, Sayer D, Wang T, Haagenson M, Spellman SR, Lee SJ, Madbouly A, Fleischhauer K, Hsu KC, Verneris MR, Thomas D, Zhang A, Sobecks R, Majhail NS

Analysis of single nucleotide polymorphisms in the gamma block of the major histocompatibility complex in association with clinical outcomes of hematopoietic cell transplantation: A CIBMTR study



Gopalakrishnan S, D'Souza A, Scott E, Fraser R, Davila O, Shah N, Gale RP, Kamble R, Diaz MA, Lazarus HM, Savani BN, Hildebrandt GC, Solh M, Freytes CO, Lee C, Kyle RA, Usmani SZ, Ganguly S, Assal A, Berdeja J, Kanate AS, Dhakal B, Meehan K, Kindwall-Keller T, Saad A, Locke F, Seo S, Nishihori T, Gergis U, Gasparetto C, Mark T, Nieto Y, Kumar S, Hari P

Revised-International Staging System (R-ISS) is predictive and prognostic for early relapse (<24 months) after autologous transplantation for newly diagnosed multiple myeloma



Lee DW, Santomasso BD, Locke FL, Ghobadi A, Turtle CJ, Brudno JN, Maus MV, Park JH, Mead E, Pavletic S, Go WY, Eldjerou L, Gardner RA, Frey N, Curran KJ, Peggs K, Pasquini M, DiPersio JF, van den Brink MRM, Komanduri KV, Grupp SA, Neelapu SS

ASBMT consensus grading for cytokine release syndrome and neurological toxicity associated with immune effector cells

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.12.75. Epub 2018 Dec 25


Page | 154


SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Bachanova V, Weisdorf DJ, Wang T, Marsh SGE, Cereb N, Haagenson MD, Spellman SR, Lee SJ, Guethlein LA, Parham P, Miller JS, Cooley S

Donor killer-cell immunoglobulin-like receptor (KIR) genotype does not improve graft-versus-leukemia responses in chronic lymphocytic leukemia (CLL) after unrelated donor transplant: A CIBMTR analysis



Pulsipher MA, Logan BR, Kiefer DM, Chitphakdithai P, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Varni JW, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Ball ED, Bolwell BJ, Bunin NJ, Cheerva A, Delgado DC, Dvorak CC, Gillio AP, Hahn TE, Hale GA, Haight AE, Hayes-Lattin BM, Kasow KA, Linenberger M, Magalhaes-Silverman M, Mori S, Prasad VK, Quigg TC, Sahdev I, Schriber JR, Shenoy S, Tse WT, Yanik GA, Navarro WH, Horowitz MM, Confer DL, Shaw BE, Switzer GE

Higher risks of toxicity and incomplete recovery in 13-17 year old females after marrow donation: RDSafe peds results



Page | 155

CIBMTR 2018 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS

APPENDIX D2: BMT CTN PUBLICATIONS

The following publications were generated by the BMT CTN, a component of the Clinical Trials Support Program, which conducts multi-institutional Phase II and III trials focused on HCT. The BMT CTN Data and Coordinating Center maintains continuity of operations and facilitates effective communications. The Data and Coordinating Center effort is a collaboration of the CIBMTR, NMDP/Be The Match, and the Emmes Corporation. For more information, see Section 2.3.1.

BMT CTN PUBLICATIONS Authors Title Citation PMCID

Pasquini MC, Logan B, Jones RJ, Alousi AM, Appelbaum FR, Bolaños-Meade J, Flowers MED, Giralt S, Horowitz MM, Jacobsohn D, Koreth J, Levine JE, Luznik L, Maziarz R, Mendizabal A, Pavletic S, Perales MA, Porter D, Reshef R, Weisdorf D, Antin JH

Blood and Marrow Transplant Clinical Trials Network report on the development of novel endpoints and selection of promising approaches for graft-versus-host disease prevention trials

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jun 1; 24(6):12741280. doi:10.1016/j.bbmt. 2018.01.002. Epub 2018 Jan 8

PMC5993573

Andermann T, Peled J, Ho C, Reddy P, Riches M, Storb R, Teshima T, van den Brink M, Alousi A, Balderman S, Chiusolo P, Clark W, Holler E, Howard A, Kean L, Koh A, McCarthy P, McCarty J, Mohty M, Nakamura R, Rezvani K, Segal B, Shaw B, Shpall E, Sung A, Weber D, Whangbo J, Wingard J, Wood W, Perales M-A, Jenq R, Bhatt A

Microbiome-host interactions in hematopoietic stem-cell transplant recipients

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jul 1; 24(7):13221340. doi:10.1016/j.bbmt. 2018.02.009. Epub 2018 Feb 19

PMC6045977

Rashidi A, Shanley R, Yohe SL, Thyagarajan B, Curtsinger J, Anasetti C, Waller EK, Miller JS, Blazar BR, Weisdorf DJ

Association between recipient TNF rs361525 and acute GVHD: Results from analysis of BMT CTN-0201 samples


PMC6064382

Page | 156


Jim HSL, Sutton S, Majhail NS, Wood WA, Jacobsen PB, Wingard JR, Wu J, Knight JM, Syrjala KL, Lee SJ

Severity, course, and predictors of sleep disruption following hematopoietic cell transplantation: A secondary data analysis from the BMT CTN 0902 trial


PMC6064383


Martens MJ, Logan BR A group sequential test for treatment effect based on the Fine-Gray model

Biometrics. 2018 Sep 1; 74(3):1006-1013. doi:10.1111/biom.12871. Epub 2018 Mar 13

PMC6146968

Spellecy R, Tarima S, Denzen E, Moore H, Abhyankar S, Dawson P, Foley A, Gersten I, Horwitz M, Idossa L, Joffe S, Kamani N, King R, Lazaryan A, Morris L, Horowitz MM, Majhail NS

Easy-to-read informed consent form for hematopoietic cell transplantation clinical trials: Results from the Blood and Marrow Transplant Clinical Trials Network 1205 study

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Oct 1; 24(10):21452151. doi:10.1016/j.bbmt. 2018.04.014. Epub 2018 Apr 18

PMC6193865

Carpenter PA, Logan BR, Lee, SJ, Weisdorf DJ, Johnston L, Costa LJ, Kitko CL, Bolaños-Meade J, Sarantopoulos S, Alousi AM, Abhyankar S, Waller EK, Mendizabal A, Zhu J, O'Brien K, Lazaryan A, Wu J, Nemecek ER, Pavletic SZ, Cutler C, Horowitz MM, Arora M

A phase II/III randomized, multicenter trial of prednisone / sirolimus versus prednisone / sirolimus / calcineurin inhibitor for the treatment of chronic graft-versushost disease: BMT CTN 0801

Haematologica. 2018 Nov 1; 103(11):1915-1924. doi:10.3324/haematol.2018.195123. Epub 2018 Jun 28

PMC6278959

Allen CE, Marsh R, Dawson P, Bollard CM, Shenoy S, Roehrs P, Hanna R, Burroughs L, Kean L, Talano JA, Schultz KR, Pai S-Y, Baker K, Andolina JR, Stenger ED, Connelly J, Ramirez A, Bryant C, Eapen M, Pulsipher MA

Reduced intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies BMT CTN 1204

Blood. 2018 Sep 27; 132(13):1438-1451. doi:10.1182/blood-201801-828277. Epub 2018 Jul 11

PMC6161764

Rashidi A, Shanley R, Yohe SL, Thyagarajan B, Curtsinger J, Anasetti C, Waller EK, Scott BL, Blazar BR, Weisdorf DJ

Recipient single nucleotide polymorphisms in Paneth cell antimicrobial peptide genes and acute

British Journal of Haematology. 2018 Sep 1; 182(6):887-894. doi:10.1111/bjh.15492.

PMC6128755

Page | 157


graftversus-host disease: Analysis of BMT CTN-0201 and -0901 samples

Epub 2018 Jul 13

Rashidi A, Wangjam T, Bhatt AS, Weisdorf DJ, Holtan SG

Antibiotic practice patterns in hematopoietic cell transplantation: A survey of Blood and Marrow Transplant Clinical Trials Network centers

American Journal of Hematology. 93(11):E348E350. doi:10.1002/ajh. 25236. Epub 2018 Jul 30

PMC6196101


Holtan SG, DeFor TE, Panoskaltsis-Mortari A, Khera N, Levine JE, Flowers MED, Lee SJ, Inamoto Y, Chen GL, Mayer S, Arora M, Palmer J, Cutler CS, Arai S, Lazaryan A, Newell LF, Jagasia MH, Pusic I, Wood WA, Renteria AS, Yanik G, Hogan WJ, Hexnar E, Ayuk F, Holler E, Bunworasate U, Efebera YA, Ferrara JLM, Pidala J, Howard A, Wu J, Bolaños-Meade J, Ho V, Alousi A, Blazar BR, Weisdorf DJ, MacMillan ML

Amphiregulin modifies the Minnesota acute graftversus-host disease risk score: Results from BMT CTN 0302/0802

Blood Advances. 2018 Aug 14; 2(15):1882-1888. doi:10.1182/bloodadvanc es.2018017343. Epub 2018 Aug 7

PMC6093743

Rashidi A, Shanley R, Anasetti C, Waller EK, Scott BL, Blazar BR, Weisdorf DJ

Analysis of BMT CTN-0201 and -0901 samples did not reproduce the reported association between recipient REG3Ars7588571 and chronic GVHD

Bone Marrow Transplantation. doi:10.1038/s41409-0180331-1. Epub 2018 Aug 31


Holstein SA, Ye JC, Howard A, Bhutani M, Gormley N, Hahn T, Hillengass J, Krishnan A, Landgren CO, Munshi NC, Olivia S, Owen RG, Pasquini MC, Puiq N, Weinhold N, Weisel K, McCarthy PL

Summary of the Second Annual BMT CTN Myeloma Intergroup Workshop on Minimal Residual Disease and Immune Profiling

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018. 11.001. Epub 2018 Nov 5


Page | 158

CIBMTR 2018 Annual Report APPENDIX D3: RCI BMT PUBLICATIONS

APPENDIX D3: RCI BMT PUBLICATIONS

The following publications were generated by the RCI BMT, a component of the Clinical Trials Support Program, which provides cellular therapy researchers with infrastructure and expertise in clinical trial conduct and analysis. For more information, see Section 2.3.2.

RCI BMT PUBLICATIONS Authors Title Citation PMCID

Nemecek ER, Hilger RA, Adams A, Shaw BE, Kiefer D, LeRademacher J, Levine JE, Yanik G, Leung W, Talano JA, Haut P, Delgado D, Kapoor N, Petrovic A, Adams R, Hanna R, Rangarajan H, Dalal J, Chewning J, Verneris MR, Epstein S, Burroughs L, Perez-Albuerne ED, Pulsipher MA, Delaney C

Treosulfan, fludarabine and low-dose total body irradiation for children and young adults with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic cell transplantation: A prospective phase II trial of the Pediatric Blood and Marrow Transplant Consortium

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Aug 1; 24(8):16511656. doi:10.1016/j.bbmt. 2018.04.025. Epub 2018 May 9

PMC6108922

Jacobsohn DA, Loken MR, Fei M, Adams A, Brodersen LE, Logan BR, Ahn KW, Shaw BE, Kletzel M, Olszewski M, Khan S, Meshinchi S, Keating A, Harris A, Teira P, Duerst RE, Margossian SP, Martin PL, Petrovic A, Dvorak CC, Nemecek ER, Boyer MW, Chen AR, Davis JH, Shenoy S, Savasan S, Hudspeth MP, Adams RH, Lewis VA, Kheradpour A, Kasow KA, Gillio AP, Haight AE, Bhatia M, Bambach BJ, Haines

HL, Quigg TC, Greiner RJ, Talano J-AM, Delgado DC, Cheerva A, Gowda M, Ahuja S, Ozkaynak M, Mitchell D, Schultz KR, Fry TJ, Loeb DM, Pulsipher MA Outcomes of measurable residual disease in pediatric acute myeloid leukemia before and after hematopoietic stem cell transplant:

Validation of difference from normal flow cytometry with chimerism studies and Wilms tumor 1 gene expression

Biology of Blood and PMC6239928 Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Oct 1; 24(10):20402046. doi:10.1016/j.bbmt. 2018.06.010. Epub 2018 Jun 19

APPENDIX D4: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS


The following publications were generated by the Health Services Research program, through which the CIBMTR identifies and addresses barriers to treatment, improves practice, and demonstrates the

Page | 159

CIBMTR 2018 Annual Report value of cellular therapies and survivorship care. For more information, see Section 2.4.

HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID

Burns LJ, Abetti B, Arnold SD, Bender J, Doughtie S, El-Jawahiri A, Gee G, Hahn T, Horowitz MM, Johnson S, Juckett M, Krishnamurit L, Kullberg S, LeMaistre CF, Loren A, Majhail NS, Murphy EA, Rizzo D, Roche-Green A, Saber W, Schatz BA, Schmit-Pokorny K, Shaw BE, Syrjala KL, Tierney DK, Ulrich C, Vanness DJ, Wood WA, Denzen EM

Engaging patients in setting a patient-centered outcomes research agenda in hematopoietic cell transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jun 1; 24(6):11111118. doi:10.1016/j.bbmt. 2018.01.029. Epub 2018 Feb 3

N/A

Khera N, Mau L-W, Denzen EM, Meyer C, Houg K, Lee SJ, Horowitz MM, Burns LJ

Translation of clinical research into practice: An impact assessment of the results from the Blood and Marrow Transplant Clinical Trials Network Protocol 0201 on unrelated graft source utilization

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Nov 1; 24(11):22042210. doi:10.1016/j.bbmt. 2018.06.028. Epub 2018 Jun 30

PMC6242749

Idossa L, Mau L-W, Ferguson SS, Denzen E, Murphy E, Moore H

Access to linguistically appropriate information for blood and marrow transplant patients: Results from transplant center staff survey

Journal of Cancer Education. doi:10.1007/ s13187-018-1407-8. Epub 2018 Aug 9

N/A

El-Jawahri A, LeBlanc TW, Burns LJ, Denzen E, Meyer C, Mau L-W, Roeland EJ, Wood WA, Petersdorf E

What do transplant physicians think about palliative care? A national survey study

Cancer. 2018 Dec 1; 124(23):4556-4566. doi:10.1002/cncr.31709. Epub 2018 Oct 5

PMC6289734

Denzen EM, Preussler JM, Murphy EA, Baker KS, Burns LJ, Foster J, Idossa L, Moore HK, Payton TJ, Haven D, Jahagirdar B, Kamani N, Rizzo JD, Salazar L, Schatz BA, Syrjala KL, Wingard JR, Majhail NS

Tailoring a survivorship care plan: patient and provider preferences for recipients of hematopoietic cell transplantation

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018. 10.005. Epub 2018 Oct 10



Page | 160

CIBMTR 2018 Annual Report HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS


Majhail NS, Murphy E, Laud P, Preussler JM, Denzen EM, Abetti B, Adams A, Besser R, Burns LJ, Cerny J, Drexler R, Hahn T, Idossa L, Jahagirdar B, Kamani N, Loren A, Mattila D, McGuirk J, Moore H, Reynolds J, Saber W, Salazar L, Schatz B, Stiff P, Wingard JR, Syrjala KL, Baker KS

Randomized controlled trial of individualized treatment summary and survivorship care plans for hematopoietic cell transplantation survivors

Haematologica. doi:10.3324/haematol.2018.203919. Epub 2018 Dec 4


Page | 161

CIBMTR 2018 Annual Report APPENDIX D5: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS

APPENDIX D5: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS

The following publications were generated by the Bioinformatics Research Program, which provides expertise in, and conducts research on, translational and operational bioinformatics. For more information, see Section 2.5.

BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID

Mack SJ, Udell J, Cohen F, Osoegawa K, Hawbecker SK, Noonan DA, Ladner MB, Goodridge D, Trachtenberg E, Oksenberg JR, Erlich HA

High resolution HLA analysis reveals independent class I haplotypes and amino-acid motifs protective for multiple sclerosis

Genes and Immunity. doi:10.1038/s41435-0170006-8. Epub 2018 Jan 8

PMC6035897

Hurley CK, Hou L, Lazaro A, Gerfen J, Enriquez E, Galarza P, Cardozo MBR, Halagan M, Maiers M, Behm D, Ng J

Next generation sequencing characterizes the extent of HLA diversity in an Argentinian registry population

HLA. doi:10.1111/tan. 13210. Epub 2018 Jan 12

N/A

Mansfield AS, Ren H, Sutor S, Sarangi V, Nair A, Davila J, Elsbernd LR, Udell JB, Dronca RS, Park S, Markovic SN, Sun Z, Halling KC, Nevala WK, Aubry MC, Dong H, Jen J

Contraction of T cell richness in lung cancer brain metastases

Scientific Reports. 8(1):2171. doi:10.1038/ s41598-018-20622-8. Epub 2018 Feb 1

PMC5794798

Roelen D, de Vaal Y, VierraGreen C, Waldvogel S, Spellman S, Claas F, Oudshoorn M

HLA mismatches that are identical for the antigen recognition domain are less immunogenic

Bone Marrow Transplantation. doi:10.1038/s41409-0180108-6. Epub 2018 Feb 6

N/A

Halagan M, Oliveira D, Maiers M, Fabreti-Oliveira RA, Moraes MEH, Visentainer JEL, Pereira NF, Romero M, Cardoso JF

The distribution of HLA haplotypes in the ethnic groups that make up the Brazilian Bone Marrow Volunteer Donor Registry (REDOME)

Immunogenetics. doi:10.1007/s00251-0181059-1. Epub 2018 Apr 26

N/A

Sivasankaran A, Williams E, Switzer GE, Cherkassky V, Maiers M

Machine learning approach to predicting stem-cell donor availability

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation.

N/A

Page | 162

CIBMTR 2018 Annual Report doi: 10.1016/j.bbmt.2018. 07.035. Epub 2018 Jul 31

APPENDIX D5: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS

BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID

Kaur N, Kransdorf EP, Pando MJ, Maiers M, Ray B, Lee J-H, Lalli P, Murphey CL, Bray RA, Gragert L

Mapping molecular HLA typing data to UNOS antigen equivalents

Human Immunology. 2018 Nov 1; 79(11):781-789. doi:10.1016/j.humimm.20 18.08.002. Epub 2018 Aug 15

N/A

Valenzuela NM, Askar M, Heidt S, Jindra P, Madbouly A, Pinelli D, Jackson A, Hidalgo LG

Minimal data reporting standards for serological testing for histocompatibility

Human Immunology. doi:10.1016/j.humimm.20 18.08.008. Epub 2018 Aug 17

N/A

Narayan S, Maiers M, Halagan M, Sathishkannan A, Naganathan C, Madbouly A, Periathiruvadi S

Human leucocyte antigen (HLA)-A, -B, -C, -DRB1 and DQB1 haplotype frequencies from 2491 cord blood units from Tamil speaking population from Tamil Nadu, India

Molecular Biology Reports. doi:10.1007/ s11033-018-4382-6. Epub 2018 Sep 18

N/A

Wang W, Huang H, Halagan M, Vierra-Green C, Heuer M, Brelsford JE, Haagenson M, Scheuermann RH, Telenti A, Biggs W, Pearson NM, Udell J, Spellman S, Maiers M, Kennedy CJ

Chromosome Y-encoded antigens associate with acute graft-versus-host disease in sex-mismatched stem cell transplant

Blood Advances. 2018 Oct 9; 2(19):2419-2429. doi: 10.1182/bloodadvances. 2018019513. Epub 2018 Sep 27

N/A

Mansfield AS, Peikert T, Smadbeck JB, Udell JBM, Garcia-Rivera E, Elsbernd L, Erskine CL, Van Keulen VP, Kosari F, Murphy SJ, Ren H, Serla VV, Schaefer Klein JL, Karagouga G, Harris FR, Sosa C, Johnson SH, Nevala W, Markovic SN, Bungum AO, Edell ES, Dong H, Cheville JC, Aubry MC, Jen J, Vasmatzis G

Neoantigenic potential of complex chromosomal rearrangements in mesothelioma

Journal of Thoracic Oncology. doi:10.1016/ j.jtho.2018.10.001. Epub 2018 Oct 10


APPENDIX D6: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS

Page | 163


APPENDIX D6: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS

The following publications were generated by the Statistical Methodology Research Program, which develops and evaluates the statistical models used in cellular therapy. For more information, see Section 2.6.

STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID

Ahn KW, Kim S Variable selection with group structure in competing risks quantile regression

Statistics in Medicine. 2018 Apr 30; 37(9):1577-1586. doi:10.1002/sim.7619. Epub 2018 Feb 21

PMC5889760

Moodie EEM, Stephens DA, Alam S, Zhang M-J, Logan B, Arora M, Spellman S, Krakow EF

A cure-rate model for Qlearning: Estimating an adaptive immunosuppressant treatment strategy for allogeneic hematopoietic cell transplant patients

Biometrical Journal. doi:10.1002/bimj.201700181. Epub 2018 May 16

PMC6239975

Wang Y, Logan BR Testing for center effects on survival and competing risks outcomes using pseudovalue regression

Lifetime Data Analysis. doi:10.1007/s10985018-9443-6. Epub 2018 Jul 5

PMC6320737

Page | 164

CIBMTR 2018 Annual Report APPENDIX D7: PUBLICATIONS NOT PREVIOUSLY REPORTED

APPENDIX D7: 2017 PUBLICATIONS NOT PREVIOUSLY REPORTED

The following publications were not presented in the 2017 CIBMTR Annual Report because they were published at the end of the year.

2017 PUBLICATIONS NOT PREVIOUSLY REPORTED Authors Title Citation PMCID (Bioinformatics Research Program)

Zhang H, Roe D, Kuang R

Detecting populationdifferentiation copy number variants in human population tree by sparse group selection

IEEE/ACM Transactions on Computational Biology and Bioinformatic. doi:10.1109/ TCBB.2017.2779481. Epub 2017 Dec 6

N/A

(Statistical Methodology Research Program) Ahn KW, Sahr N, Kim S

Screening group variables in the proportional hazards model

Statistics & Probability Letters. 2018 Apr 1; 135:2025. doi:10.1016/j.spl.2017. 11.014. Epub 2017 Dec 13

PMC6051756

(BMT CTN) Smith EP, Li H, Friedberg JW, Constine LS, Rimsza LM, Cook JR, Laport GG, Popplewell LL, Holmberg LA, Smith SM, LeBlanc M, Forman SJ, Fisher RI, Stiff P

Tandem autologous hematopoietic cell transplantation for patients with primary progressive or recurrent Hodgkin lymphoma: A SWOG and Blood and Marrow Transplant Clinical Trials Network phase II trial (SWOG S0410/BMT CTN 0703)

Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Apr 1; 24(4):700-707. doi:10.1016/j.bbmt.2017.12.798. Epub 2017 Dec 28

PMC5965270

APPENDIX E: PRESENTATIONS

2018 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETINGStudy Title Type PI

BMT CTN 0402 Effect of sirolimus on immune reconstitution following myeloablative allogeneic stem-cell transplantation: A posthoc analysis of a randomized controlled trial comparing sirolimus / tacrolimus with tacrolimus / methotrexate (BMT CTN 0402)

Poster M Gooptu

CK15-01 Survival advantage to allogeneic transplant in patients with myelofibrosis with intermediate-1 or higher DIPSS score

Poster K Gowin K Bellen R Mesa

Page | 165

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS CK16-02b Tyrosine kinase inhibitors with or without donor lymphocyte

infusion continue to provide long-term survival after relapse of chronic myeloid leukemia following hematopoietic cell transplantation

Oral S Schmidt

CK18-01 A personalized prediction model for outcomes after allogeneic stem cell transplant in patients with myelodysplastic syndromes

Oral A Nazha

DS17-01 Weighty choices: Selecting optimal G-CSF doses for stem cell mobilization to optimize yield

Oral N Farhadfar

GS17-02 T-replete haploidentical cell transplantation using post-transplant cyclophosphamide for acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndrome: Effect of transplant conditioning regimen intensity on outcomes

Oral S Soloman

GV16-02 Impact of T-cell dose on GVHD risk after allogeneic HLA-matched PBSC transplantation

Poster A Saad S Hashmi M Sharma

L Lamb HS15-01 Lost to follow-up rates are higher in pediatric than adult

survivors, but not by transplant type: A report from the Center for International Blood and Marrow Transplant Research

Poster D Buchbinder

HS15-02 Area-based socioeconomic status and pediatric allogeneic hematopoietic stem cell transplantation outcomes: A CIBMTR analysis

Oral K Bona

HS16-02 The impact of marital status on hematopoietic stem cell transplant recipient outcomes: A surrogate for consistent caregiver. A CIBMTR registry study

Poster S Beattie

2018 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI

HSR-1608 Health care reimbursement and service utilization among Medicare beneficiaries with multiple myeloma receiving autologous hematopoietic cell transplantation in inpatient and outpatient settings

Oral N Dunavin

IB14-03c Telomere length and telomerase complex mutations predict fatal treatment toxicity after stem cell transplantation in patients with myelodysplastic syndrome

Oral R Coleman

IB15-03 Outcomes of pediatric patients with acute leukemia following adult unrelated donor transplant: The impact of donor KIR gene content and KIR ligand matching

Poster M Verneris J Miller

S Cooley

Page | 166

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS IB15-07 Multiple functional donor polymorphisms in IL1RL1 region

associate with death due to GVHD or infection after unrelated donor allogeneic hematopoietic stem cell transplantation for AML and MDS

Oral S Paczesny S Spellman J Renbarger

IB16-01 The role of HLA-E compatibility in the prognosis of acute leukemia patients undergoing 10/10 HLA matched unrelated HSCT

Oral C Tsamadou D Furst

J Mytilineos

IB16-03 Donor IFNL4 genotype is associated with transplant-related mortality after unrelated donor myeloablative hematopoietic cell transplantation in patients with acute leukemia

Oral S Gadalla L

ProkuninaOlsson

IN16-02 Burden and outcomes of mucosal barrier injury-laboratory confirmed bloodstream infections in the first 100 days after allogeneic stem cell transplant: A CIBMTR analysis

Poster C Dandoy

LE13-020 Risk factors for the development of cutaneous melanoma after allogeneic hematopoietic cell transplantation

Oral M Herr

LE17-02 Impact of myeloablative total body irradiation versus chemotherapy on late effects and survival among adolescent and young adult survivors of hematopoietic cell transplantation for acute leukemia: A CIBMTR analysis

Oral C Lee L Muffly

LK15-01 Allogeneic hematopoietic cell transplantation vs non-HCT consolidation therapies in acute myeloid leukemia patients 60-75 years of age in first complete remission: An Alliance (A151509), SWOG, ECOG-ACRIN and CIBMTR study

Poster A Artz C Ustun

LK16-01 Reduced intensity conditioning regimens hematopoietic cell transplantation for acute myeloid leukemia: A comparison of fludarabine / busulfan and fludarabine / melphalan based regimens from the CIBMTR

Poster Z Gul H Alkhateeb

R Nath

2018 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI

LY17-03 Impact of allogeneic hematopoietic cell transplantation on the outcomes of angioimmunoblastic T-cell lymphoma: A CIBMTR analysis

Oral N Epperla

NM17-02 Related and unrelated donor transplantation for β thalassemia major: Results of an international survey

Oral V Mathews

RT09-04b / IB09-06

Genome wide association analyses identify pleitropic variants associated with AML and MDS susceptibility

2018 BMT TANDEM MEETINGS

Poster L SuchestonCampbell

Page | 167

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS Study Title Type PI

BMT CTN 1203 Novel approaches for graft-versus-host disease prophylaxis: Primary results of PROGRESS I multicenter trial of matched allogeneic hematopoietic cell transplantation using reduced intensity conditioning: BMT CTN 1203

Oral J BolañosMeade

GV14-01 Graft-versus-host disease prophylaxis in reduced intensity conditioning allogeneic hematopoietic cell transplantation: Comparing the efficacy of mycophenolate mofetil and methotrexate in combination with calcineurin inhibitor

Poster S Chhabra

GV14-01 Cyclosporine in combination with mycophenolate mofetil leads to increased incidence of graft-versus-host disease and inferior outcomes after myeloablative allogeneic hematopoietic cell transplantation: A CIBMTR analysis

Poster B Hamilton

HSR-1603 A national survey study of transplant physicians’ attitudes about palliative care

Oral A El-Jawahri

HSR-1605 Estimating propensity scores for the receipt of allogeneic hematopoietic cell transplantation in outcomes research using claims data: A machine learning approach

Poster D Vanness

IB09-06/ RT0904b

Genome-wide significant donor genetic associations with disease death in AML and MDS patients in the first 1 year after BMT are not modified by conditioning intensity or TBI

Oral T Hahn

IB09-06/ RT0904b

Genetic associations with day +100 transplant related mortality after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY-BMT study)

Oral T Hahn

IB10-01c Clonal alterations and survival after unrelated donor allogeneic hematopoietic stem cell transplant in patients with Fanconi anemia

Oral Y Wang

2018 BMT TANDEM MEETINGSStudy Title Type PI

IB15-01 Analysis of single nucleotide polymorphisms donor / recipient mismatches in the gamma block of the major histocompatibility complex and their association with hematopoietic cell transplantation outcomes: A CIBMTR study

Poster M Askar

IB15-02 Graft-versus-leukemia responses in chronic lymphocytic leukemia are not influenced by natural killer cell KIR immunogenetics: A CIBMTR analysis

Oral V Bachanova

IN14-01 Outcomes of allogeneic hematopoietic cell transplants with EBV positive or EBV negative post-transplant lymphoproliferative disorder

Poster S Naik

Page | 168

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS IN16-01 Viral isolates in the cerebrospinal fluid of hematopoietic stem

cell transplant recipients Poster M Abidi

LE16-03 Survivors of childhood allogeneic hematopoietic cell transplant have higher unemployment rates compared to the general US population

Oral N Bhatt

LY17-01 Outcome of patients 65 years and older with non-Hodgkin lymphoma receiving reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age: A CIBMTR analysis

Oral N Shah

RT07-01b Evaluation of the hematopoietic cell comorbidity index (HCTCI) in recipients of allogeneic transplantation for nonmalignant diseases

Oral L Brogile

RT14--01 Survival trends after allogeneic hematopoietic cell transplant in children less than one-year-old

Oral S Parikg

RT14-03 Improved mortality prognostication for critically ill pediatric hematopoietic cell transplant patients: Results from a Virtual Pediatric Systems and CIBMTR database merger

Oral M Zinter

Bioinformatics The marked expansion of hematopoietic stem and progenitor cells with aryl hydrocarbon receptor antagonist in MGTA-456 significantly improves the probability of identifying a 5-6/6 HLA-matched umbilical cord blood donor

Poster M Maiers


Phase II trials and the use of registry controls Oral R Brazauskas

2018 EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION

ANNUAL MEETING Program Title Type PI

CK16-02a Contemporary role of maintenance tyrosine kinase inhibitors following allogeneic hematopoietic cell transplantation for chronic myeloid leukemia: A CIBMTR analysis

Oral Z DeFilipp

DS16-S2 Survey of the screening and management for clonal disorders of hematopoiesis in related allogeneic donors

Poster M Seftel

LE16-02 New malignant neoplasms after alloHCT for pediatric patients with non-malignant diseases

Oral J Kahn

RT07-01b Co-morbidities / aging impact on HCT for non-malignant diseases

Oral M Thakar

SC17-02 Veno-occlusive disease characteristics in pediatric patients with acute myeloid leukemia receiving gemtuzumab ozogamicin before allogeneic stem cell transplant

Poster C Duncan

Page | 169

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS SC17-02 Characterization of veno-occlusive disease in adult patients

with acute myeloid leukemia receiving gemtuzumab ozogamicin before allogeneic stem cell transplant

Poster V Ho

2018 DATA STANDARDS AND SYMPOSIUM HACKATHON Program Title Type PI

Bioinformatics Introduction to FHIR: HL7, resources, profiles, versions, and ballots

Oral B Milius

Bioinformatics Genomics reporting implementation guide Oral B Milius

Bioinformatics Tool for high throughput conversion of HML to FHIR Oral A Brown

Bioinformatics Laboratory reporting sequences to curation service: Allele calling tool

Oral M Halagan

Bioinformatics HLA terminology services Oral J Schneider

Bioinformatics Beyond FHIR to a genomic data repository Oral Y Bolon

2018 AMERICAN SOCIETY OF HISTOCOMPATIBILITY AND IMMUNOGENETICS ANNUAL MEETING

Program Title Type PI

Bioinformatics A method for assessing immunogenicity of HLA-epitopes to circumvent potential immunotoxicity of therapeutic gene editing

Oral W Wang

Bioinformatics Landscape of CIBMTR/NMDP research capabilities provided through Bioinformatics services

Oral Y Bolon

Bioinformatics A community resource using gene feature enumeration to generate accurate allele calls and sequence annotations for HLA and KIR

Poster M Halagan

Bioinformatics The common uncommon extended HLA haplotype: Do two weak associations make it strong?

Poster MZ Askar

Immunobiology Limited exon sequence mismatches outside the antigen recognition domain in a cohort of 4,646 high resolution 10/10 HLA-matched donor and recipient pairs

Poster H Huang

2018 EUROPEAN FEDERATION FOR IMMUNOGENETICS ANNUAL CONFERENCEProgram Title Type PI

Bioinformatics Single haplotype admixture models using large scale HLA Oral Y Louzoun

Page | 170

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS genotypes to reproduce human admixture A Litniski

A Madbouly M Halagan M Maiers

Bioinformatics High resolution HLA-A, -B, -C, -DRB1, and –DQB1 Allele and haplotype frequencies for Arab Donors in the Hadassah bone marrow donor registry

Poster A Bishara M Halagan C Brautbar

S Israel M Maiers

A Madbouly Bioinformatics HLA Class I Haplotype Diversity is Consistent with Selection for

frequent Existing Haplotypes Poster Y Louzoun

L Gragert M Maiers

I Alter S Fingerson

2018 AMERICAN SOCIETY OF CLINICAL ONCOLOGY ANNUAL MEETING

Study Title Type PI

BMT CTN 1506 A Phase 3, trial of gilteritinib a maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with FLT3-ITD+ AML

Poster MJ Levis

HSR-1603 A national survey study of transplant physicians’ attitudes Poster A El-Jawahri about palliative care

2018 EASTERN NORTH AMERICAN REGION INTERNATIONAL BIOMETRIC SOCIETY SPRING MEETING



Adaptive group bridge for marginal Cox proportional hazards Oral models with multiple diseases

N Sahr S Kim

KW Ahn


Competing risks regression for case-cohort design Oral

2018 NMDP/BE THE MATCH COUNCIL MEETING

S Kim Y Xu

MJ Zhang KW Ahn

Study Title Type PI

DS13-01 The quality of harvested bone marrow for transplantation Oral has decreased over time: Implications and solutions

N Prokopishyn

Page | 171

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS DS16-01

One versus two day apheresis in unrelated donors Oral 2018 ACADEMY HEALTH ANNUAL RESEARCH MEETING

J Hsu

Study Title Type PI

HSR-1501 Healthcare reimbursement and service utilization for first Poster E Denzen year post-allogeneic hematopoietic cell transplantation among Medicare beneficiaries ages 65 and older with acute myeloid leukemia

2018 AMERICAN SOCIETY OF PEDIATRIC HEMATOLOGY/ONCOLOGY ANNUAL

CONFERENCE

Study Title Type PI

DS16-S1 Transplant center practices for psychosocial assessment and Oral G Switzer management of pediatric HSC donors

2018 CANADIAN BLOOD AND MARROW TRANSPLANT GROUP ANNUAL MEETING Study Title Type PI

DS16-S2

Survey of the screening and management for clonal disorders of hematopoiesis in related allo donors

2018 HLA AND KIR POPULATION DYNAMICS WO

Poster

RKSHOP

M Seftel


Bioinformatics HLA and KIR: Selection and admixture Oral M Maiers

2018 JOINT STATISTICAL MEETINGS OF THE AMERICAN STATISTICAL ASSOCIATION Program Title Type PI


Analysis of competing risk data in generalized case-cohort design

2018 KIR WORKSHOP

Oral Y Xu


Immunobiology Patterns of KIR-HLA gametic linkage disequilibrium in a large Oral C Vierra-Green U.S.

Page | 172

CIBMTR 2018 Annual Report APPENDIX E: PRESENTATIONS transplantation cohort

2018 NATIONAL SICKLE CELL DISEASE ASSOCIATION OF AMERICA ANNUAL MEETING


Health Availability of information on blood and marrow Oral T Mupfudze Services transplantation coverage for patients with sickle cell disease enrolled

in Medicaid

Page | 173

CIBMTR 2018 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS

APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS

This study development and management process pertains to studies for which the CIBMTR provides data, scientific, and statistical support. Data sets are also made available to investigators who have their own statistical resources. Final analyses and manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission.

STUDY DEVELOPMENT AND MANAGEMENT PROCESS

Planned Protocol pending. Proposals remain in this preliminary stage until the principal investigator (PI) creates a draft protocol.

In Progress

Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it.

Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee.

Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome.

Supplemental forms / data collection. Most studies use routinely-collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on centers and supplement forms reimbursement costs.

Page | 174



In Progress (continued)

Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow-up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS-level statistician, working with the Scientific Director, PI(s), and sometimes the Clinical Research Coordinator, to ensure data quality:

• Verifying selection criteria • Including and excluding patients so that the investigators can determine whether the final

study population is representative of the target population • Assessing follow-up • Determining the extent and nature of missing values and their potential effect on the study • Resolving and reconciling data discrepancies / outliers by examining data collection forms and

communicating with centers and the PI

Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Study PI(s) and associated Working Committee Chairs present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript.

Ongoing. A study in ongoing status is long-term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study typically has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year.

Preliminary Results

Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. Study Leadership reviews and revises the document, ensuring that the description and interpretation of the statistical analyses are accurate and contribute to the fundamental message of the manuscript. The Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript.

Page | 175



Preliminary Results

(continued)

Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician forwards all editor and reviewer comments to the PI and Statistical Director. The PI is expected to prepare a response, working with Study Leadership who provide additional analyses of data, as needed. Coordinating Center personnel communicate with the journal, including re-submissions, in most cases.

In press. A publication is in press when it has been approved but does not yet have a citation.

Completed Published. A manuscript is considered published when a citation is available, including a PMCID number, if applicable. For a list of this year’s publications, see Appendix D.

Page | 176

CIBMTR 2018 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS

APPENDIX G: CLINICAL STUDIES AND TRIALS

Through the Clinical Trials Support Program, the Coordinating Center supports clinical trial planning and interpretation; data collection, including long-term follow-up data; and real-time accrual assessment. See Section 2.3 for more information.

APPENDIX G1: BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT

The BMT CTN (Section 2.3.1) is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation. A status of BMT CTN trials open for enrollment is included in this appendix and is available on the BMT CTN website. For additional information on completed Network trials, see the annual progress report on the BMT CTN website.

BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Number

Title Status to Date

BMT CTN 1201

(Alliance A051301)

A randomized double-blind Phase III study of ibrutinib during and following autologous stem cell transplantation versus placebo in patients with relapsed or refractory diffuse large B-cell lymphoma of the activated B-cell subtype

• Opened to accrual Jul 2016 • 31 of 296 patients enrolled • Anticipated accrual completion in mid

2020

BMT CTN 1502 Optimizing cord blood and haploidentical aplastic anemia transplantation (CHAMP)

• Opened to accrual May 2017 • 13 of 30 patients enrolled • Anticipated accrual completion in mid

2020

BMT CTN 1503 A study to compare bone marrow transplantation to standard care in adolescents and young adults with severe sickle cell disease

• Opened to accrual Nov 2016 • 73 of 200 patients enrolled • Anticipated accrual completion in late

2020

BMT CTN 1506 A multi-center, randomized, double-blind, placebo-controlled Phase III Trial of the FLT3 inhibitor gilteritinib administered as maintenance therapy following allogeneic transplant for patients with FLT3/ITD AML

• Opened to accrual May 2017 • 142 of 346 patients enrolled • Anticipated accrual completion in late

2019

BMT CTN Reduced intensity conditioning for 1507 haploidentical bone marrow

Page | 177

CIBMTR 2018 Annual Report transplantation in patients with symptomatic sickle cell disease

• Opened to accrual Oct 2017

• 25 of 80 patients enrolled • Anticipated accrual completion in

late 2021 APPENDIX G1: BMT CTN CLINICAL TRIALS

BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT

Protocol Title Status to Date

Number

BMT CTN 1601

(ECOG-ACRIN EA4151)

A randomized Phase III trial of consolidation with autologous HCT followed by maintenance rituximab vs. maintenance rituximab alone for patients with mantle cell lymphoma in minimal residual disease-negative first complete remission

• Opened to accrual Aug 2017 • 42 of 689 patients enrolled • Anticipated accrual completion in mid

2021

Page | 178

CIBMTR 2018 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES

APPENDIX G2: RCI BMT CLINICAL STUDIES

The RCI BMT (Section 2.3.2) provides researchers in the field of cellular therapy with infrastructure and expertise in clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting into such groups as the BMT CTN or the national cancer cooperative groups. It also facilitates large survey and cohort studies. A status of its projects is included in this appendix.

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Protocol Number


11-TREO Multi-center study evaluating treosulfan, fludarabine, and low-dose TBI in children with AML / MDS undergoing allogeneic HCT

• Closed to accrual April 2014 • 40 pediatric recipients enrolled • Abstracts presented at 2016 EBMT and

2016 BMT Tandem Meetings • Manuscript published Aug 2018

06-DON RDSafe: A multi-institutional study of hematopoietic stem cell donor safety and quality of life

• Closed to accrual July 2014 • 1,812 donors enrolled • Follow up assessments completed July 2015 • Ancillary study manuscript published June 2017;

primary manuscript and two ancillary study manuscripts published in 2018 (Oct, Nov, Dec)

09-MRD A multi-center study to determine the role of minimal residual disease testing before and after HCT for pediatric acute myeloid leukemia

• Closed to accrual Oct 2014 • 150 pediatric recipients enrolled • Manuscript published Oct 2018

09-PLEX A Phase II study evaluating the safety and efficacy of intravenous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematological malignancies

• Closed to accrual Dec 2014 • 128 enrolled (64 recipient-donor pairs) • Follow-up completed Feb 2016 • Manuscript pending resubmission

13-TLEC Prospective non-therapeutic study, assessing the long-term toxicity of HCT for childhood leukemia

• Closed to accrual Mar 2018 • 340 recipients enrolled; 31 enrolled in 2018

PBSC Filgrastim-mobilized peripheral blood stem cells for allogeneic transplantation with unrelated donors

• Opened to accrual Apr 1996 • >38,000 unrelated donors enrolled

CIBMTR 2018 Annual Report RCI BMT CLINICAL STUDIES

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CIBMTR 2018 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES RCI BMT CLINICAL STUDIES

Protocol Number


10-CBA A multi-center access and distribution protocol for unlicensed cryopreserved cord blood units for transplantation in pediatric and adult patients with hematologic malignancies and other indications

• Opened to accrual Oct 2011 • 4,118 recipients enrolled; 401 enrolled in 2018 • Open indefinitely to allow access and distribution

of unlicensed cord blood units

BMT CTN 1102-QOL

A study comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome

• Survey Research Group performing quality of life assessments

• 225 total assessments completed in 2018

BMT CTN 1102-

Ancillary CEA study

A cost effectiveness ancillary study to parent study 1102-QOL above

• Collaboration with Fred Hutchinson Cancer Research Center

• Survey Research Group performing cost effectiveness analysis (CEA) survey collection

• First subject contacted in Oct 2015 • 83 surveys completed in 2018

13-SCP A randomized study to evaluate the impact of survivorship care planning on cancer survivors’ self-management and adherence to care recommendations and utilization of follow-up care

• Collaboration with Health Services Research Program

• Closed to accrual June 2016 • 495 recipients enrolled • Manuscript accepted for publication Nov 2018

MMP Millennial member project: Survey Research Group supporting Be The Match Operations project

• First subject contacted by the Survey Research Group in July 2016

• 1,957 registry members / donors completed the study

15-MMUD HLA-mismatched unrelated donor bone marrow transplantation with post-transplantation cyclophosphamide for patients with hematologic malignancies

• Opened to accrual Aug 2016

• 176 of 80 recipients enrolled; 34 enrolled in 2017

State Street Generation of an induced pluripotent stem cell bank immune matched to a majority of the US population

• First donors contacted Jan 2017 • 8 of 12 whole blood products collected

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CIBMTR 2018 Annual Report RCI BMT CLINICAL STUDIES

Protocol Number


17-PRO Investigate quality of life in older vs younger patients receiving transplants for myelodysplasia

• Companion study to CIBMTR CMSapproved expanded access study

• Serves as pilot study for new ePRO system • Opened to accrual June 2016 • 24 subjects enrolled in 2018

17-CSIDE Busulfan exposure-finding for SCID • Opened to accrual Oct 2018 • 1 of 64 subjects enrolled

17-SIBS Identifying predictors of poor healthrelated quality-of-life among pediatric hematopoietic stem cell donors

• Initial NMDP IRB approval Aug 2017 • Study pending first accrual

16-NTCD Naïve T cell depletion for prevention of chronic GVHD in pediatric population • Initial protocol team meeting Oct 2016

• Initial DSMB approval July 2017 • Initial NMDP IRB approval Nov 2017 • Study pending first accrual in 2019

17-CD33- CAR-T

Phase 1 study of CD33-redirected chimeric antigen receptor T cell immunotherapy in children and young adults with relapsed or refractory acute myeloid leukemia

• Protocol team established June 2017 • Pre-IND meeting with FDA Nov 2018

INSPIRE-SCP Integrating health informatics in a scalable stepped care selfmanagement program for HCT survivors

• Collaboration with Fred Hutchinson Cancer Research Center

• Initial protocol team meeting Feb 2017

• RCI BMT will prepare individualized care plans and contact patients for electronic surveys

APPENDIX H: FORMS SUBMISSION PROCESS

APPENDIX H: FORMS SUBMISSION PROCESS

• Center submits CRID Assignment Form (Form 2804), and CRID is generated • Indication for CRID Assignment Form (Form 2814) is added to Forms Due list • Center completes Indication Form and reports indication as HCT • Pre-TED (Form 2400) is added to Forms Due list • Center completes and submits Pre-TED

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CIBMTR 2018 Annual Report APPENDIX G2: RCI BMT CLINICAL STUDIES • Pre-TED data are processed through the selection algorithm resulting in CRF or TED track o If autologous

recipient declines consent for research, stop here. Otherwise, follow the appropriate track below

CRF Track TED Track

1 Forms 2004, 2005, and 2006 are added, depending on donor type and if the donor has been used for a prior transplant.*

Forms 2004, 2005, and 2006 are added, depending on donor type, consent for sample repository, and if the donor has been used for a prior transplant.*

2 Baseline form 2000, disease specific inserts, and Followup Forms are added to Forms Due list.

Post-TED Follow-up Form 2450 is added to Forms Due list.

3 Center completes Baseline form after infusion. Center completes designated Post-TED Forms at appropriate time points.

4 Center completes designated CRF Follow-up Forms at appropriate time points.

Is recipient alive? If yes, go to Step 5. If no, report the death on the follow-up form, and go to Reporting Recipient Death.

5 Is recipient alive? If yes, go to Step 6. If no, report the death on the follow-up form, and go to Reporting Recipient Death.

Did recipient have a subsequent transplant? If yes, go to Step 6. If no, continue reporting at next time point (Step 3).

6 Did recipient have subsequent transplant? If yes, go to Step 7. If no, continue reporting at next time point (Step 4).

Subsequent transplant is reported on the next available follow-up form.

7 When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.

When the form reporting the subsequent transplant is in complete status, future forms for the prior transplant will be automatically deleted from FormsNet.

8 Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.

Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant.

Reporting Recipient Death

Death Form 2900 is completed to report the recipient’s death.**

The recipient’s death is reported on the Post TED. A 2900 Death Form should not be completed for patients on the TED track.

* For more details regarding when Forms 2004, 2005, and 2006 are required, see “How Forms Come Due (2004, 2005, and 2006)”.

**Complete Death Form 2900 even if autopsy is pending. Another death form will be requested to confirm cause of death if autopsy was pending.

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CIBMTR 2018 Annual Report APPENDIX I: WEBSITES

APPENDIX I: WEBSITES

Throughout this report, electronic links to webpages and documents are provided; they are underlined and italicized for identification. If you are unable to access items using the links provided, enter the underlined and italicized words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). URLs for the websites mentioned in this report are provided here.

Name URL

Be The Match bethematch.org Be The Match Clinical bethematchclinical.org BMT CTN bmtctn.net CIBMTR cibmtr.org CIBMTR Collaborate collaborate.cibmtr.org CIBMTR Portal portal.cibmtr.org HRSA CWBYCTP bloodcell.transplant.hrsa.gov

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CIBMTR 2018 Annual Report APPENDIX J: GLOSSARY

APPENDIX J: GLOSSARY

Abbreviation/ Meaning Acronym

AGNIS A Growable Network Information System alloHCT allogeneic hematopoietic cell transplantation AML acute myeloid (myelogenous) leukemia ASBMT American Society of Blood and Marrow Transplantation ASH American Society of Hematology BMT CTN Blood and Marrow Transplant Clinical Trials Network BRIDG Biomedical Research Integrated Domain Group BSI bloodstream infection CAR chimeric antigen receptor CDE common data elements CDISC Clinical Data Interchange Standards Consortium CED Coverage with Evidence Determination CIBMTR Center for International Blood and Marrow Transplant Research CIDR Cellular Immunotherapy Data Resource CITI Collaborative IRB Training Initiative CMS Centers for Medicare and Medicaid Services CPI Continuous Process Improvement CRF Comprehensive Report Form CRID CIBMTR Recipient Identification Number CTED Cellular Therapy Essential Data CWBYCTP C.W. Bill Young Cell Transplantation Program DBtC Data Back to Centers application DISCO Data and Information for Statistical Center Operations DRI Disease Risk Index EAIN Engagement Award Initiative Notice EBMT European Society for Blood and Marrow Transplantation eDBtC enhanced Data Back to Centers application ePRO electronic patient-reported outcomes FACT Foundation for the Accreditation of Cellular Therapy FDA Food and Drug Administration FHIR Fast Healthcare Interoperability Resources GDPR General Data Protection Requirement of the European Union GVHD graft-versus-host disease HCT hematopoietic cell transplantation HIPAA Health Insurance Portability and Accountability Act

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CIBMTR 2018 Annual Report HLA human leukocyte antigen HRSA Health Resources and Services Administration IBMTR International Bone Marrow Transplant Registry IND Investigational new drug IOTN Immuno-Oncology Transplantation Network

APPENDIX J: GLOSSARY

Abbreviation/ Meaning

Acronym

IRB Institutional Review Board IT Information Technology KIR killer-cell immunoglobulin-like receptors MCW Medical College of Wisconsin MDS myelodysplastic syndrome MUD matched unrelated donor N/A not applicable NCI National Cancer Institute NHLBI National Heart, Lung, and Blood Institute NIAID National Institute of Allergy and Infectious Disease NIH National Institutes of Health NMDP National Marrow Donor Program PBMTC Pediatric Blood and Marrow Transplant Consortium PBSC peripheral blood stem cell PCORI Patient-Centered Outcomes Research Institute PI principal investigator PMCID PubMed Central unique identifier PNH paroxysmal nocturnal hemoglobinuria PRO patient-reported outcomes PROMIS Patient Reported Outcome Measurement Information System QOL quality of life RCI BMT Resource for Clinical Investigations in Blood and Marrow Transplantation RDSafe Related Donor Safety Study REMS risk evaluation and mitigation strategy SCTOD Stem Cell Therapeutic Outcomes Database SNP single nucleotide polymorphisms TED Transplant Essential Data US United States VOD veno-occlusive disease vs versus WBMT Worldwide Network for Blood and Marrow Transplantation WMDA World Marrow Donor Association

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The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID).

CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and Medical College of Wisconsin.

cibmtr.org(763) 406-5800Minneapolis, MN 55401 USA

Streetth5 N 500 Be The Match National Marrow Donor Program/Minneapolis Campus

cibmtr.org(414) 805-0700Milwaukee, WI 53226 USA

5500 W. Wisconsin Ave., Suite C9200 Medical College of WisconsinMilwaukee Campus

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