Overview of Stem Cell Transplant

A.KewBlood Matters

October 29, 2010

Disclosures• Financial disclosures

–None• Advisory Boards

–Novartis

ObjectiveProvide an overview of Bone Marrow

Transplantation

SCT Timeline

Principles of SCT

• Allows the administration of high-dose therapy

• Takes advantage of graft-versus-leukemia effect

Principles of SCT

• Source of stem cells– Autologous

– Allogeneic• HLA-matched sibling• Matched, unrelated donor• Umbilical cord blood

– Cells• Bone marrow• Peripheral blood

Bone marrow

• 750-1500mL harvested from posterior iliac crests– general anesthesia– generally well tolerated

• NMDP– 6 deaths from normal

donors– 1/10,000 risk

Peripheral Blood

• CD34+ cells circulate in the peripheral blood– Increase following

administration of chemotherapy and/or growth factors

• NMDP– 3 deaths amongst PB

donors

Indications for SCT

• Hematologic malignancies

• Other– Solid tumors – Immunodeficiencies– Autoimmune diseases– Hemoglobinopathies

What type of transplant?

AllogeneicAplastic anemiaAutologousMyelomaAllogeneicCLLAutologous/AllogeneicIndolent lymphomaUsually autologousHodgkin lymphomaUsually autologousAggressive lymphomaAllogeneicMDSAllogeneicCMLAllogeneicALLAllogeneicAML

Indications for Transplant

3,000

5,500

2,000

1,500

5000

1,000

2,500

3,5004,0004,5005,000

Indications for hematopoietic stem cell Indications for hematopoietic stem cell transplant in North Americatransplant in North America

20082008

SUM10_11.pptSlide 9

Num

ber

of

Tra

nsp

lants

MultipleMyeloma

NHL AML HD ALL MDS/MPD AplasticAnemia

CML OtherLeuk

OtherCancer

Non-Malig

Disease

Allogeneic (Total N=6,672)

Autologous (Total N=10,302)

Indications for TransplantIndications for allogeneic hematopoietic stem Indications for allogeneic hematopoietic stem

cell transplantation in North Americacell transplantation in North America20082008

SUM10_13.pptSlide 11

3,000

2,000

1,500

500

0

1,000

2,500

Num

ber

of

Tra

nsp

lants

MultipleMyeloma

NHLAML HDALL MDS/MPD AplasticAnemia

CML OtherLeuk

OtherCancer

Non-Malig

Disease

Related Donor (Total N=3,282)

Unrelated Donor (Total N=3,389)

What type of transplant?

5-10%Non-myeloablative

30-40%MUD

20%Sibling allogeneic

5-10%Autologous

Risk of TRMType of transplant

Transplant Complications

• Acute complications– Infections

– Acute graft-versus-host disease

– VOD

Acute GVHD

• Clinical manifestations– Rash

– Diarrhea

– Hepatic dysfunction

VOD

• Clinical features– sudden weight gain

– hepatomegaly (tender)

– jaundice

– peripheral edema and ascites

Long-term Complications• Toxicities from treatment

regimens

• Immune deficiency

• Autoimmune syndromes

• Infectious complications

• Endocrine disturbances

• Chronic GVHD

• Second malignancies

• Cognitive dysfunction

• Psychosocial adjustment

• Decreased quality of life

Causes of Death

Causes of death Causes of death after transplantations after transplantations

done in 2003done in 2003--20082008

SUM10_22.pptSlide 20

Infection (17%)Other Cause

(19%)

Organ Failure (12%)

Primary Disease(35%)

IPn* (5%)

GVHD (12%)Infection(14%)

Other (22%)

GVHD (10%)

Primary Disease (43%)

IPn* (3%)

Organ Failure (8%)

Infection (5%)

Other Cause (17%)

Organ Failure (4%)

IPn* (1%)Primary Disease (73%)

*IPn = Idiopathic pneumonia syndrome

AutologousAutologous

Unrelated donorUnrelated donor

HLAHLA--identical siblingidentical sibling

Donor Selection

• HLA match• Age• Sex• CMV status• ABO group

Questions?