Agenda 8:00pm Welcome Marc Richmond / Elfi Pahl 8:10pm Steering Committee Report Marc Richmond 8:20pm DCC Update James Kirklin / Nick Timkovich 8:40pm Database Committee Report Jennifer Conway 8:55pm Quality of Life Update Karen Uzark 9:00pm Foundation Update Craig Collum 9:10pm Scientific Committee Report Elfi Pahl 9:20pm Enduring Hearts Bob Boucek 9:25pm New Foundation Officers Melanie Everitt 9:30pm Adjourn

Thank you to our sponsor! 4/4/2019

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. 4/4/2019

1993 2003 2013 Today Institutions 21 25 46 56* Countries 1 2 3 4 PHTS Continues to Grow 1993 2003 2013 Today Institutions 21 25 46 56* Countries 1 2 3 4 Listings 236 2513 6695 8901 Transplants 140 1736 4816 6562 Abstracts - 26 87 123 Publications 9 57 86 Newest Hospitals Enrolled: UCSF-Benioff Children’s Hospital and Children’s Hospital MN

2017 Pediatric Transplants US Hospitals (50) 431 Reported by UNOS for all pediatric heart transplant centers 400 Reported by UNOS for PHTS Centers 93% Potential percentage of all US pediatric heart transplants if all PHTS centers report 100% of individual center transplants* 378 Reported to PHTS for the calendar year 2017 88% of US transplants (no informed consent, not eligible) *some patients may be ineligible

Building a Better Dataset Steps for rigorous 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

Building a Better Dataset Inclusion Criteria for Research Datasets: 90% Rules 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 2018: 100% of active centers compliant

Building a Better Dataset Form revisions 1993, 1996, 1999, 2005, 2010, 2015 2015: Web Based Data Entry System Launch DCC Data Manager checking all variables across all form revisions Major mapping issues / form changes VAD and ECMO previously on listing and transplant form, moved to MCSD form Revisions to inclusion criteria Estimated Completion of Mapping Project: Spring 2019

Data Submission Deadlines Event Occurrence Months Absolute Submission Deadline 1st Quarter January February March April 30 2nd Quarter April May June July 31 3rd Quarter July August September October 31 4th Quarter October November December January 31

Quality Assurance Reports Reports including events through June 30, 2018 will be available December 2018

New Reporting Features in PHTS Wed-Based Data Entry Nick Timkovich Director of Database Development, Management & Reporting UAB KIRSO 4/4/2019

PHTS Reporting Features Overview Data Download (2019) Sites can retrieve all data in bulk that they own in the WBDE Clinical Summary Report (2019) Single PDF of all details of a single patient Cohort Comparison (2019) Compare multiple cohorts of patients for specific data elements frequencies Outcome Analytics (Future) Survival Curves, Competing Outcomes, etc. based upon user-selected cohorts 4/4/2019

PHTS Reporting Features Data Download Form by form download of all data entered by the site Excel files that contain: Common information, such as Patient identifiers, site identifiers, system columns (unique identifiers for each form, tracking information, etc.) ALL data from the form 4/4/2019

PHTS Reporting Features Data Download 4/4/2019

PHTS Reporting Features Clinical Summary Report PDF file of clinical data for a single patient. All relevant information for that patient in one place. Phase 1: Single button to open from Dashboard or Patient Summary Customizable charts Expand/Collapse sections 4/4/2019

PHTS Reporting Features Cohort Comparison Report PDF of basic summary data for User-selected Cohorts Bar charts for frequencies, tables of raw data Three options: Single Cohort ex: Males, Age at Listing = 1 to 6 months, Cardiomyopathy Two Cohorts within your site ex: Males vs Females, Age at Transplant = 1 year to 6 years. Single Cohort comparing your site to the aggregate ex: NYHA Class 1 at your center vs all centers 4/4/2019

PHTS Reporting Features Outcomes Analytics Advanced Analytics based on user-selected criteria, generating charts, graphs, and other elements to convey outcome-related information Kaplan Meier Survival Curves Competing Outcomes Risk-adjusted Models Other options to be determined Real-time generation of reports (PDF) based on checked data Tailored to needs of the PHTS community Future release of PHTS, depending on input from leadership and community 4/4/2019

PHTS Reporting Features THANK YOU 4/4/2019