PHTS: Building a Better Dataset for Pediatric Heart Transplantation Research

1. Agenda

2. Thank you to our sponsors: Thank You!

3. UAB DCC Personnel

4. 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.

5. PHTS Continues to Grow *210 Unique co-authors since 1993

6. Data Collection and Submission

7. 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

8. 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)

9. 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.

10. 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

11. Data Submission Deadlines First bi-annual Quality Assurance (QA) reports to be distributed in December 2017.

12. Quality Assurance Reports Reports will contain data on events through June 30, 2017.

13. Quality Assurance Reports ALL 53 active centers will be included in reports for the first time in 5 years! *PHTS current has 54 participating centers. 53 active centers and 1 inactive center.

14. Data Submission Deadlines

15. Quality Assurance Reports Reports including events through December 31, 2017 will be available July 2018

16. Quality Assurance Should PHTS Be a Quality Assurance Registry, which could avoid missing patients? CurrentPractices • 23 centers (43%) have a waiver of informed consent • 31 centers (57%) require informed consent 2016 Listings • 40 (6%)patients declined consent in 2016 • 583 Listings entered Reasons for No Consent: • Declined Consent • Language Barrier • Died before consent • Too sick to consent

17. Web Based Data Entry System Recent Releases

18. Version 1.1.2 (Released August 9, 2017) Compliance Reporting • Users can see in real time their missing and completed follow-up forms in relation to their compliance score for follow-ups for all time, the previous year, and the current year. • Users can download their annual follow-up reports which details all follow-ups submitted for their site. • Users can see in real time their follow-up percentage to help meet quarterly deadlines and ensure site stays above 90% compliance as we approach the form submission deadlines.

19. Version 1.1.3 (Released September 29, 2017) Sticky Notes • Sticky notes are now used for all data integrity checks. • Sites can see all data integrity checks on their dashboard all in one place with their form submission (previously all done through email) • Streamlines data inquiries being sent back to the sites. • Users can view reports of sticky notes sent to determine common issues with data submission. • More efficient data cleaning allows for better quality data. • Sites can add their own sticky notes to send to colleagues if they have questions about how a particular form should be completed.

20. Web Based Data Entry System Upcoming Releases 1.1.4 Release (Will Be Available December 2017) • Clinical trial tracking • Revised infection definition • Infection organism mapping Upcoming Releases • Annual follow-up medication mapping (previously free-text)* • Listing and transplant reconciliation added to wbde system, • Additional range checks and in-form validations (including lab values and hemodynamics) • Enhanced patient summary • Past due form rules • Data Download (presented at April meeting) • Analytics (presented at April meeting) • Clinical Summary Report (presented at April meeting)

21. Web Based Data Entry System Upcoming Releases 1.1.4 Release (Will Be Available December 2017) • Clinical trial tracking • Revised infection definition • Infection organism mapping Upcoming Releases • Annual follow-up medication mapping (previously free-text)* • Listing and transplant reconciliation added to wbde system, • Additional range checks and in-form validations (including lab values and hemodynamics) • Enhanced patient summary • Past due form rules • Data Download (presented at April meeting) • Analytics (presented at April meeting) • Clinical Summary Report (presented at April meeting)

22. WBDE: Analytic Software Package Data Download • The Form Download is a tool that allows sites to download all data they have entered into the Web-Based Data Entry System. • The Form Download is broken down by each individual form. The Form Download follows the Web-Based Data Entry Screen • Allow sites to download any data they enter into the Web-Based Data Entry by form in Excel format • Allow sites to see an instantaneous update from data entered in the Web-Based Data Entry to each downloaded form

23. Excel Charts from the Form Download (Intermacs) Implant Year (2015) by Device Type Implant Year (2016) by Device Type

24. WBDE: Analytic Software Package Clinical Summary Report • Provide coordinators with an easy to read summary on all clinical characteristics for a single patient • Key information in the PHTS Web-Based Data Entry condensed into a few summary pages • Contains charts and other helpful features to see changes in lab vales over time

25. Clinical Summary – De-identifed Example (Intermacs)

26. WBDE: Analytic Software Package Cohort Comparison • The Cohort Comparison tool entails the creation and delivery of reports that describe the user-defined cohorts. After the user has selected the criteria for the cohort, they may select “cohort report” and print a PDF report of their selection • The user-defined cohorts may be compared: • A single cohort selection from a site • Between the site’s own patients based on subgroups (cohort # 1 and cohort # 2) • Between the site’s own patients (cohort # 1) and an equivalent overall PHTS (cohort # 2)

27. Example: Competing Outcomes (Intermacs)

28. Example: Subgroups Within Site - Kaplan-Meier (Intermacs)

29. Web Based Data Entry System Analytic Software Package (available in Jan. 2019) • Data Download • Clinical Summary Report • Cohort Comparison • Outcome Analytics