ATHN Data Summit Thrombosis Data Collection: Lessons Learned from the HTRS TERegistry

1. ATHN Data SummitThrombosis Data Collection: Lessons Learned from the HTRS TERegistry Bryce A. Kerlin, MD Director, Hemostasis & Thrombosis Center Nationwide Children’s Hospital Assistant Professor of Pediatrics The Ohio State University College of Medicine

2. Topics • HTRS TERegistry: • Registry Objectives • Data Collection • Quality Control / Data Integrity • Accrual and Enrollment Limitations • ATHN Specific Issues: • Limitations of Data Quantity • Role of ATHN in Thrombosis Research • Protection of Investigator(s)

3. TERegistry Obejectives • Prospectively evaluate the epidemiology and clinical characteristics of the known prothrombotic risk factors in persons with TE by developing a clinical informatics database (“registry”). • Identify the frequency and nature of complications associated with TE and its treatment. • Describe the phenotypes and complications seen in persons with multiple molecular risk factors. • Compare the epidemiology, clinical characteristics, and complications seen in persons with and without known risk factors. May be better described as a “natural history” study.

4. Inclusion / Exclusion • Inclusion: • Patient’s first TE. • DVT, Arterial, Intra-Cardiac Thrombi; PE; Arterial TE; Stroke (<20 yrs); MI (<20 yrs). • Specific criteria for diagnosis must be met. • Exclusion: • Bleeding Disorders; TIA; Sickle Cell; Metabolic Disorders associated with Stroke; Hemorrhagic Stroke; Bacterial Endocarditis; MAHA.

5. Data Entry • Paper Data Worksheets • For medical record abstraction • Worksheets • Broken down into data types (registration, lab data, etc.) • Worksheets faxed to main study center (NCH/OSU) • Data Transcribed into secure database • Quality Control • Worksheets archived at NCH and at primary institution

6. Data Collection Flow

7. Typical Worksheets

8. Database Screenshots

9. Database Screenshots

10. Quality Control / Data Integrity • Data Entry • Single person enters data • Quarterly Data Survey • 6 random charts audited for accurate data entry on a quarterly basis • 3 new registrations / 2 follow-ups / 1 recurrence • Database • Nightly Backup • Password Protected • Encrypted • Access Limited to Study Personnel

11. Enrollment • Current: 110 patients <20 years old • Adult enrollment much slower • Goal: 500 patients <20 years old

12. Adult Enrollment Barriers • Late presentation to study center • Limited Diagnostic Data available • Less frequently tested for thrombophilia • Epidemiology / Natural History already better defined • Lack of Research Assistant Support / Infrastructure

13. Pediatric Enrollment Issues

14. Pediatric Enrollment Issues • Other: • Debate regarding utility of thrombophilia testing

15. ATHN Specific Issues

16. D C C B B B A A A A MD MD MD MD A B C D MD MD MD MD Data Quantity • “UDC Style” • Epidemiology Driven Database / Registry • “COG / Alternate Style” • Hypothesis Driven Database / Registry E E

17. ATHN Role in Thrombosis Research • Facilitator? • i.e. Data Warehouse • Provides means to collect data, distribute data, and provider of research tools • Statistical support • Scientific Review • Prioritization • Scientific Body? • i.e. Study Group • Development of Hypotheses • Study Design • Both?

18. Investigator Protection • Massive Time and Effort • Intellectual Property • Data integrity must be preserved for the initiating investigator(s) utilization • Examples: • HTRS TERegistry • Co-owned by Kerlin, NCH, HTRS • CDC Pilot • Co-owned by CDC and Pilot Centers