Annual Spring Meeting April 5, 2017

1. Annual Spring MeetingApril 5, 2017

2. Thank you to our sponsors: Thank You!

3. PHTS Purpose (Est. 1993) The purposes of the group are to establish and maintain an international, prospective, event driven database for heart transplantation, to use the database to encourage and stimulate basic and clinical research in the field of pediatric heart transplantation and to promote new therapeutic strategies..(An additional current focus is QA and QI)

4. UAB DCC Personnel

5. PHTS Continues to Grow

6. PHTS Continues to Grow • 53 participating PHTS hospitals • 50 hospitals represented here tonight • 5 non PHTS hospitals, several in process of joining • 156 registered meeting attendees

7. PHTS Participating Hospitals United States (48 hospitals)

8. PHTS International53 Hospitals • United States • Canada • Brazil • England

9. Data Collection and Submission

10. Data Submission Deadlines

11. Building a Better Dataset Steps for data entry and checking • Work closely with coordinators to ensure timely and complete data submission • Regularly scheduled data consistency checks • Work with coordinators to correct errors or enter missing forms • Check number of transplants reported to UNOS by institution for US hospitals and with PIs for non-US hospitals

12. Building a Better Dataset Inclusion Criteria ALL pediatric patients listed for heart transplantation on or after the date of study entry for an institution are eligible for inclusion in the study. Re-listed patients can now be enrolled at the new PHTS center as a new patient. (As of 09/01/2015) Simultaneous organ transplantation (other than combined heart-lung) is no longer an exclusion criterion. (As of 01/01/2010)

13. Building a Better Dataset Exclusion Criteria Patients who are 18 years of age or greater at the time of listing. (NOTE: there is no age restriction on patient follow-up once they are enrolled. The age restriction is strictly at time of listing) Patients who are listed for a combined heart-lung transplant.

14. Building a Better Dataset Inclusion Criteria for Research Datasets • 90% compliant with all listing forms • 90% compliant with all transplant, donor, and immunosuppression forms • 90% compliant with all annual follow-up forms • 90% compliant with all event forms • Addressed all data queries • In process of incorporating these compliance scores into the data entry system for real time visualization

15. Quality Assurance Reports QA Reports containing data through 12/31/2016 will be available June 2017

16. 2016 Pediatric Transplants US Hospitals (47)

17. 2016 Pediatric Transplants Non-US Hospitals (5)

18. International Expansion Upcoming Booths • ISHLT • April 5-7, 2017 • World Congress • July 16-21, 2017 (Barcelona, Spain) PHTS is working to continue to expand the database globally.

19. DCC Update: Research

20. 2017 Research Proposals Abstracts and Presentations • ISHLT 2017 (Abstract Deadline: October 25, 2016) • 6 abstracts accepted • 4 analyses at UAB • 2 limited data set requests • 56 unique co-authors (representing 29 hospitals) • AHA 2017 (Abstract Deadline: June 12, 2017) • 2 proposals accepted • all analyses at UAB • 14 unique co-authors (representing 10 hospitals) • ISHLT 2018 Proposals under review - (Abstract Deadline: October/November 2017) • 3 proposals to be accepted • all analyses at UAB • 20 unique coauthors (representing 16 hospitals) 372 total members of PHTS (including both investigators and coordinators)

21. PHTS Membership Writing Group Distribution Publications and Abstracts: 1995-2017 • 76 Publications: 51 Unique First Authors • 177 Total Unique co-authors • 111 Abstracts: 78 Unique First Authors • 254 Total Unique co-authors

22. Impact of PHTS Research on Heart Transplant Community

23. Research Challenges • Submitting few papers at time of meeting • Unintended consequences: • Slow submission leads to risk of data becoming out of date with resultant request for “updated” analyses • Invades time needed for current analyses and abstract development • Promotes a culture of lack of discipline in designing (at the outset) the original study in a way that accurately and completely drives the analytic plan

24. Research Challenges • Submitting few papers at time of meeting • Unintended consequences: • Slow submission leads to risk of data becoming out of date with resultant request for “updated” analyses • Invades time needed for current analyses and abstract development • Promotes a culture of lack of discipline in designing (at the outset) the original study in a way that accurately and completely drives the analytic plan • ISHLT 2017: 0/6 submitted at time of ISHLT • Expect 6/6 will be submitted immediately after ISHLT

25. Limited Data Set Requests Policy and Procedures • Purpose to increase scientific productivity and to make data available to members • De-identified dataset specific to the approved proposal is provided to investigators for one analysis, abstract, and manuscript • Goal of PHTS to provide • High quality research • Research equity to all members in good standing who actively participate in data submission

26. Limited Data Set Requests Submitted Requests • Announced to membership September 25, 2013 • Limited data set sent to requesting center • Analyses performed at requesting center • Fee for request: $5,000 • 2013 submitted request: 1 • 2014 submitted request: 4 • 2015 submitted request: 3 • 2016 submitted request: 1 • 2017 submitted request: 0

27. Collaboration with UAB • We remain committed to the purposes and vision of the PHTS. • We see a bright future for this 24 year collaboration. • We are always interested in ideas to improve the clinical relevance and productivity of the PHTS and our collaboration.