Annual Fall Meeting November 13, 2016 6:30pm Welcome J. Kirklin/M. Everitt 6:40pm Steering Committee Report M. Everitt 6:50pm DCC Update J. Kirklin 7:05pm Web Based Data Entry D. Naftel 7:20pm Database Committee Report M. Richmond 7:35pm Foundation Update C. Collum/B. Wilk/R. Gajarski 7:50pm Risk Factor Model K. Schumacher 8:05pm Heart Failure Simulation B. Feingold 8:10pm iPHFR R. Gajarski 8:15pm Break 8:30pm Pedimacs Report B. Blume 8:40pm Scientific Committee Report D. Hsu 8:50pm Scientific Committee Sponsored Workshops 9:35pm New Business 9:45pm Adjourn

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. So, this is the mission statement for PHTS, established in 1993. Note how closely it mirrors our current activities.

UAB DCC Personnel James K. Kirklin, MD KIRSO Division Director David Naftel, PhD Program Director and Statistician Craig Collum, MPH Foundation Executive Director Susanna Lenderman Program Manager Elizabeth Pruitt, MSPH Analytic Manager Lauren Turner Project Coordinator Camesha Grigsby Data Entry Operator Brittney Wilk Development Director Monica Henderson Chase Lenderman Website Administrator Additional Support Nick Timkovich Lead Developer (WBDE) Brandon Sharp Developer (WBDE) Ramaraju Rudraraju, PhD John Pennington Programmer Susan Myers Ryan Cantor, MSPH David Helms, MA Devin Kohl Grant Studdard The PHTS research efforts have a large group at UAB to draw upon for database and research analytics expertise, many of whom are available for this effort because of a majority of their funding from other sources.

General Report of the DCC Hospital Growth Enrollment History of Current PHTS Hospitals This chart depicts the growth in hospital participation over the past 23 years. So, note that we’ve grown from 15 hospitals in 1993 to 52 in 2016…

PHTS Participating Hospitals United States (47 hospitals) With wide representation from all over the US…You’ll note the carefully constructed color scheme to make sure that we don’t infer any political statement.

PHTS World Wide 52 Hospitals (+2 in process) United States Canada Brazil England Japan (in process) Australia (interested) With additional programs in Canada ( list programs) , one in Brazil ( list) , and one in England (list). We also have a document that is nearly completed from a consortium in Japan and interest from a center in Australia ( list) Blue=Participating hospitals Green=Interested hospitals

Abstracts and Presentations 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) We have had a productive year in abstract presentations….read.

Impact of PHTS Research on Heart Transplant Community This is a depiction of presentations ( red) and public (blue) Discuss #s: 2005-2010- 5 presentations/yr, 4public’s/yr; 2010-14 - 6/yr pres, 5/yr pubs; recently 7-12 pres/yr, 7-8 pub/yr

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 Impressive overall track record in abs/pubs

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 However, we continue to have challenges in turning presentations into publications in a timely fashion. So, our goal is always to turn in a manuscript at the time of presentation. The importance of this relates directly to our ability to efficiently move other projects into the cue . …

Limited Data Set Request 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 An important strategy to increase the number of studies and publications is outsourcing limited or “de-identified” data sets. Discuss Limited Data Sets ( make sure this is de-identified data sets) . Daphne Hsu will discuss in more detail

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 Limited Data Sets

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) MENTORSHIP PROGRAM. Is this a quote from Gajarski? What year did this start?

Investigator Visits to UAB 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 Find out which of these were de-identified data sets sent out.

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 CHECK WHERE JENN CONWAY IS

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 Check this- a prior slide indicates that there was 1 in 2016? Are these approved?

Looking Ahead: 2017 Goals Research Proposals AHA ISHLT 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

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. Finally , just to reaffirm the commitment of UAB….