Annual Fall Meeting November 13, 2016

1. Annual Fall MeetingNovember 13, 2016

2. 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.

3. UAB DCC Personnel

4. General Report of the DCCHospital Growth Enrollment History of Current PHTS Hospitals

5. PHTS Participating Hospitals United States (47 hospitals)

6. PHTS World Wide52 Hospitals (+2 in process) • United States • Canada • Brazil • England • Japan (in process) • Australia (interested) Blue=Participating hospitals Green=Interested hospitals

7. 2016: Year in Review Abstracts and Presentations ISHLT 2016, April, Washington DC • 10 presentations • 5 analyses at UAB • 5 limited data set requests • 60 unique co-authors (representing 29 hospitals) AHA 2016, November, New Orleans • 2 presentations (analyses at UAB) • 15 unique authors (representing 13 hospitals) ISHLT 2017: Abstract Deadline October 25, 2016 • 6 abstracts submitted • 4 analyses at UAB • 2 limited data set requests • 46 unique co-authors (representing 28 hospitals)

8. Impact of PHTS Research on Heart Transplant Community

9. PHTS Membership Writing Group Distribution Publications and Abstracts: 1995-2016 • 70 Publications: 51 Unique First Authors • 177 Total Unique co-authors • 109 Abstracts: 73 Unique First Authors • 256 Total Unique co-authors

10. 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• Submitting few papers at time of meeting • Unintended consequences: - Slow submission leads to risk of data becoming out of date with resultant request for “updated” analyses - Invades time needed for current analyses and abstract development - Promotes a culture of lack of discipline in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

11. 2016 Year in Review Limited Data Set Request • 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

12. Limited Data Set Request 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

13. Scientific Committee Mentorship Program “More experienced participants on this committee will bring the required skillset to successfully conduct the research studies evidenced either by advanced training in health outcomes research and/or an established track record of high quality peer reviewed publications in journals such as AJT, Circulation, JHLT, etc. This will facilitate the development of high quality project proposals and learning by those with less experience.” - Rob Gajarski, PHTS Past President, Strategic Redesign of Scientific Investigation through the PHTS (2015)

14. 2016 Year in Review • Investigator Visits to UAB • January 11-12: Melanie Everitt, Daphne Hsu (Leadership Visit) • February 19: Kurt Schumacher & Charlie Canter (Risk Factor Model) • March 22: Bob Morrow, Joshua Sparks (Diabetes Post Transplant) • March 28: Bibhuti Das (Donor Crossmatch) • September 27: Jennifer Conway, Elfi Pahl (ECMO vs VAD <10kg) • September 28: David Sutcliffe, Betsy Blume

16. ISHLT 2017 Abstract Submission Analyses at UAB • Mechanical Circulatory Support (MCS) in Infants with Congenital Heart Disease listed for heart transplant - A PHTS Multicenter Experience Jennifer Conway, MD Stollery Children’s Hospital, University of Alberta • Post-Transplant Outcomes in Pediatric VAD Patients: A Pedimacs-PHTS Linkage Study David Sutcliffe, MD Children’s Health Dallas, University of Texas Southwestern • The long-term outcomes of patients supported with the Berlin Heart EXCOR as a bridge to transplantation: A multi-institutional study Aamir Jeewa, MD Hospital for Sick Children • Association of Insurance Coverage on the Development of Coronary Allograft Vasculopathy after Pediatric Heart Transplant Elizabeth Pruitt, MSPH University of Alabama at Birmingham

17. ISHLT 2017 Abstract Submission Limited Data Set Request • HLA Eplet Mismatching Is Associated with Increased Risk of Graft Loss in Pediatric Heart Transplant Recipients Erin Albers, MD Seattle Children’s Hospital • Hemodynamics of the Pediatric Failing Fontan John Dykes, MD Lucile Packard Children’s Hospital at Stanford

18. Looking Ahead: 2017 Goals Research Proposals AHA • Proposal Deadline: October 2016 • Abstract Submission Deadline: June 2017 • Number of proposals to be accepted: 2 • Number of writing group members to be assigned: 10 • Number of centers to be represented:10 ISHLT • Proposal Deadline: March 2017 • Abstract Submission Deadline: October/November 2017 • Number of proposals to be accepted: 4 • Number of writing group members to be assigned: 20 • Number of centers to be represented: 20 Note: The DCC has agreed to analyze 6 proposals per year

19. 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.