development of ccfa partners kids & teens: an internet-based cohort of pediatric ibd michael d....

Development of CCFA Partners Kids & Teens: an Internet-Based Cohort of Pediatric IBD

Michael D. Kappelman Wenli ChenChristopher F. Martin Beth JaegerErin Stoeber Lucy GobleTania Kamphaus Robert S. SandlerMillie D. Long

DISCLOSURES

Nothing to disclose

Background

Prospective cohorts of pediatric IBD are needed to:• Study disease natural history• Identify prognostic factors and/or environmental risk factors of relapse• Evaluate of the safety and effectiveness of treatments

Most pediatric IBD cohorts in the U.S. rely on center-based recruitment and data collection• Involve patients followed at large and/or academically oriented practices• Very time and resource intensive• Focus on physician-reported data and biosample collection

CCFA Partners is an Internet-based cohort of adults with IBD• Large and diverse study population: >12,000 patients from all 50 states,

treated in academic and private practice settings• Web-based recruitment and data collection efficient and cost-effective• Focus on patient reported exposures, health behaviors, and outcomes

Objective

To develop an internet based cohort of pediatric IBD patients in collaboration with the Crohn’s and Colitis Foundation of America

Methods

• Recruited children (< 18 years of age) with self-reported IBD through email invitations to CCFA constituents, promotion on the CCFA website, social media, and chapter events

• After informed consent/assent, parents and their children completed a baseline web-based survey containing questions regarding:

• Demographics• Disease characteristics (hospitalization, surgery,

anthropometrics, location, extent)• Disease activity (Patient Global Assessment, Short CDAI,

PUCAI, and Manitoba Index)• Treatments• Adherence• Quality of life (Impact 35) and other Patient Reported

Outcomes

Methods 2

• Surveys developmentally appropriate, with increasing levels of self-report based upon age

• Validated survey instruments used when available • At 3 month intervals, an update on study progress and

educational materials developed by CCFA emailed to participants

• Follow up surveys planned Q6 months• Enrollment and linkage to adult CCFA Partners planned upon

18th birthday

www.ccfapartners.org

Preliminary Findings (First 4 months)

Enrollment:• 570 Participants from 47 U.S. states and 6 countries• 316 male (55%), 254 female (45%)• 422 CD (74%), 134 UC (24%), 14 IC (2%)• Median age: 13 years• Median age at diagnosis: 9 years

Treatment Patterns

CD n=422 UC n=148

Current Medications

N (%) N (%)

5-ASA (oral) 117 (27) 57 (38)

Prednisone 41 (9) 23 (15)

Immunomodulator 186 (43) 52 (34)

Biologic 220 (51) 28 (18)

Medications ever-used

5-ASA (oral) 251 (58) 116 (76)

Prednisone 354 (82) 129 (85)

Immunomodulator 294 (68) 84 (55)

Biologic 249 (57) 57 (38)

Surgery 71 (16) 26 (17)

Patient Reported Outcomes

1 2 3 430

40

50

60

70

Mean PROMIS domain scores by IMPACT Quartile*

Depressive Symptoms

Anxiety

Peer relations

Fatigue

Pain Interference

IMPACT Quartile

*Higher IMPACT scores indicate better HRQOL

Patient Reported Outcomes

Remiss

ion (

sym

ptom

-free

)

A Few

Sym

ptom

s

Sympt

oms

Somet

imes

A Lot

of S

ympt

oms

Sympt

oms

All the

Tim

e30

40

50

60

70

Mean PROMIS domain scores by patient global assessment

Depressive Symptoms

Anxiety

Peer relations

Fatigue

Pain Interference

Patient Global Assessment

Conclusion

CCFA Partners Kids & Teens is a novel internet-based cohort of pediatric IBD• Partnership between CCFA, patient community, and research

community• Channel for all children with IBD to participate in research,

regardless of treating physician/practice• Preliminary findings suggest that internet based recruitment and

data collection are feasible• Growth in enrollment and long term cohort retention will be critical to

the success of this project

Implications and future directions

Focus on PROs will become increasingly important in observational research• Complement to clinical endpoints, which are rapidly moving towards

mucosal healing rather than measures of patient well being

Dual enrollment and linkage with clinical cohorts (i.e. Risk, Protect) will allow analyses that combine clinician-reported data, patient-reported data, and biospecimens

Long term outcomes of pediatric IBD• Opportunity for follow up after patients transition from pediatric care

Platform for ancillary studies• Secondary analyses, supplemental surveys, simple clinical trials,

biospecimen collection

Please Get involved

Submit ancillary studies

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