1. CCFA Partners: An Internet-based Cohort Study Robert S. Sandler, MD, MPH University of North Carolina Chapel Hill, NC

2. Outline Introduction – name, vision, who we are How it works Enrollment/retention What we have learned so far Sharing the results Ancillary studies Validation study Biospecimen feasibility New directions Summary

3. The name CCFA IBD patient community UNC research team

4. CCFA partners research team Robert Sandler, MD, MPH, PI Michael Kappelman, MD, MPH, co-PI Millie Long, MD, MPH, co-PI Kristen Anton, informatics director Chris Martin, MPH, epidemiologist Wenli Chen, programmer Beth Jaeger, project manager Lucy Goble, research assistant

5. Our vision Use Internet-based recruitment and data collection to enroll and follow a large, diverse IBD cohort ●Efficient and inexpensive Focus on patient-reported data: ●Exposures (i.e. diet, medications, smoking) ●Health behaviors (i.e. adherence, prevention) ●Outcomes (i.e. fatigue, depression, anxiety, pain) Provide a platform for ancillary studies: observational, interventional, translational

6. How it works Enrolled >12,000 IBD patients from CCFA email rosters, CCFA web- page link, social media, walks, etc. Baseline survey ●Core module (medications, surgery, disease activity) ●Optional modules 6-month surveys to determine change in treatment or health status (optional modules) Use 3-month contacts to deliver educational messages ●Reminder about prevention activities such as flu vaccine, etc. ●Enhance participation

7. Core data Baseline and Q6 month follow-up Health care utilization Smoking IBD hospitalizations, surgery, cancer Medications NSAIDs Family history of IBD IBD history Current disease activity Prevention Exercise Quality of life-SIBDQ Patient reported outcomes-PROMIS

8. Three month messages

9. Progress

10. Enrollment

11. Source of self-registered patients

12. Follow-up time

13. Maintaining participation

14. Publications

15. Papers (7) Cohort development Dietary patterns Geriatric depression Perceptions of care Patient reported outcomes Sleep Vaccine beliefs Abstracts (22) Cohort development Prevention Adherence Quality of life Diet Patient reported outcomes Perceptions of chronic care Depression Biobanking Gluten Behavioral interventions Immunizations Update on cohort Prevention messages Validation Exercise Sleep Pregnancy Hormones Kids & Teens NSAIDS Email prevention messages *submitted

16. What we have learned so far Prevention - About half had a bone density scan or took calcium or vitamin D supplements. Only 40% reported having a skin exam and 16% reported always wearing sunscreen. Less than 2/3 had an flu vaccine. Adherence – majority had adherence in low range; disease activity correlated with low adherence Quality of life – worse QOL with more disease activity; better QOL in UC with colectomy PROMIS - IBD patients had more anxiety, depression, fatigue and sleep disturbance, and less social satisfaction than the general population. Using corticosteroids made all of the outcomes worse. Elderly depression - as many as 1 in 4 elderly patients with IBD may suffer from depression. Depressed elderly patients with IBD were more likely to have more severe disease activity and less likely to take their medication correctly all of the time Sexual interest - women, patients with disease around their rectum and patients with more active disease had less sexual interest and satisfaction. Half of patients with ostomies said that their ostomy affected their sexual satisfaction.

17. Sharing results Infographics Lay summaries

18. Ancillary studies General categories ●Analyses of existing data ●Supplemental surveys (fertility, adverse med effects, infections) distributed to all participants or a subset (females only, age- restricted, prior surgery, etc) ●Educational interventions – ‘ask your doctor about a flu shot’ ●Recruitment for other studies (biospecimens, chart extraction, RCT) ●Combination of above Varying assistance by DMC ●Turn-key (form design, data management, data analysis, etc) ●Independent investigator (DMC assists with subject selection, recruitment, prepares extract of CCFA Partners data) ●Hybrid

19. Ancillary studies Ashwin Ananthakrishnan, MGH, Bidirectional Association Between Sleep and Disease Activity (abstract and accepted paper) Frank Farraye, BU, Patient Vaccine Survey (abstract) Joshua Korzenik, BWH, An Investigation into Diet and its Role with Exacerbations of Ulcerative Colitis Gill Melmed, Cedars-Sinai, Sex Hormones on Disease Activity (abstracts) Maria Ferris, UNC, The Role of Health Literacy, Social Support, Social Media and ADHD symptoms in Predicting Medication Adherence Lawrence Gaines, Vanderbilt, Depression as a Risk Factor for Crohn’s Balfour Sartor, UNC, Genetic Associations for Rare IBD phenotypes

20. FACES Food and Crohn’s Exacerbation Study James Lewis

21. Validity “On the Internet, nobody knows you’re a dog.”

22. CCFA Partners-GSK Validation Study Aim: To assess the validity of self-reported medical data in the CCFA Partners internet cohort Method: Compare specific aspects of self-reported IBD characteristics to information from treating physician, including: ●Confirmation of IBD (not IBS or diverticulitis) ●Type of IBD (Crohn’s disease or ulcerative colitis) ●Location or extent of disease ●Crohn’s disease behavior (stricturing or penetrating) ●Extra-intestinal disease manifestations ●Prior surgeries ●Current pouch or ostomy

23. CCFA Partners-GSK Validation Study Compare physician information to participant self-reported information Physician completes Validation Form online or by mail Physician mailed Validation Form Participant consents and provides information on treating physician Participant mailed information, consent and HIPAA forms CCFA Partners participant expresses interest in study

24. CCFA Partners-GSK Validation Study

25. 3884 indicated interest 450 invited to participate 258 consented (57%) 160 physician reports obtained (62%) 5 no IBD (3%) 155 confirmed IBD (97%) 7 disease type did not match (5%) 148 disease type matched (95%) 92 not returned 4 physicians refused 2 physicians left practice 139 not returned (31%) 47 refused (10%) 6 ineligible

26. Biospecimens Biorepository pilot ●Assess willingness of respondents to provide genetic samples or other biospecimens ●Establish infrastructure to collect, ship, store, and analyze genetic and fecal biospecimens ●Compare feasibility and recruitment rates for different methods of sample collection

27. Biospecimens

28. New directions Data collection beyond surveys Enhanced Patient Engagement ●Increased enrollment ●Improved retention

29. Digital future

30. mHealth use by CCFA Partners Cohort

31. mHealth app and device

32. Enhanced patient engagement ●Sharing of Research Findings ●Involving Patients in Prioritization of Research Agenda ●Patient Functionality ●Social Networking/Community Building

33. Summary ●Harness the power of the Internet to efficiently enroll and follow unprecedented numbers of IBD patients ●Largest US study of the impact of IBD. Already enrolled subjects from all 50 states, 4 U.S. territories, urban and rural, cared for by private and academic GI’s ●Help to advance methods in patient reported outcomes for clinical research and epidemiology ●Platform to recruit for other studies (interventional, epidemiological, HSR, translational) ●Beyond research, has the potential to impact patient lives through prevention messages and disease education.

34. Summary