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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB0191 - myGTR: a patient engagement tool from the World Federation of Hemophilia Gene Therapy Registry

    Sunday, June 25, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Co-Author(s)

    • Toong Youttananukorn, PhD

      Data Quality Manager, Gene Therapy Registry
      World Federation of Hemophilia
      Montreal, Quebec, Canada

      • Co-Author(s)

    • Barbara A. Konkle, MD (she/her/hers)

      Professor of Medicine
      University of Washington
      Seattle, Washington, United States

    • Donna Coffin, MSc Epid (she/her/hers)

      Director, Research & Education
      World Federation of Hemophilia
      Montreal, Quebec, Canada

      • Presenting Author(s)

    • MN

      Mayss Naccache, MSc

      Gene Therapy Program Manager
      World Federation of Hemophilia
      Montreal, Quebec, Canada

      • Co-Author(s)

    • CC

      Cary Clark

      Director of Programs and Education
      International Society on Thrombosis and Haemostasis
      Carrboro, North Carolina, United States

    • Lindsey A. George, MD

      Assistant Professor of Pediatrics
      University of Pennsylvania
      Philadelphia, Pennsylvania, United States

    • AI

      Alfonso Iorio

      Professor
      Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON
      Hamilton, Ontario, Canada

    • Wolfgang Miesbach, MD

      Professor and Head of Coagulation Disorders and Comprehensive Care Centre
      University Hospital Frankfurt, Frankfurt, Germany
      Frankfurt, Hessen, Germany

    • DN

      Declan Noone

      President
      European Haemophilia Consortium
      Brussels, Brussels Hoofdstedelijk Gewest, Belgium

    • Flora Peyvandi, MD, PhD (she/her/hers)

      Director
      IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
      Milano, Lombardia, Italy

    • Steven W. Pipe, MD

      Professor of Pediatrics and Pathology
      University of Michigan, Ann Arbor, MI, USA
      Ann Arbor, Michigan, United States

    • Michael Recht, MD, PhD, MBA (he/him/his)

      Chief Medical and Scientific Officer
      National Hemophilia Foundation
      New York, New York, United States

    • Mark W. Skinner, JD (he/him/his)

      President and CEO
      Institute for Policy Advancement Ltd.
      Washington, District of Columbia, United States

    • LV

      Leonard A. Valentino

      Physician
      Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois, USA
      New York, New York, United States

    • Johnny Mahlangu, MD (he/him/his)

      Professor and Director
      Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
      Johannesburg, Gauteng, South Africa

    • Glenn F. Pierce, MD PhD

      VP Medical
      World Federation of Hemophilia
      Montreal, Quebec, Canada

    Background: Collection of long-term data to monitor safety and efficacy is crucial to ensure the well-being of people with hemophilia (PWH) who receive Gene Therapy (GT). These clinical data will be collected by clinicians via the WFH Gene Therapy Registry (GTR). Patient-reported outcome (PRO) data will be collected to complement these data and to address a potential data gap if PWH decrease their visits to their HTC after GT.

    Aims: To fill this potential gap the WFH is developing myGTR – a patient engagement tool aimed at capturing PRO data.

    Methods: The WFH held focus groups with patient advocates (N = 9, [received GT, N = 5; not received GT, N = 4]), healthcare providers (N = 9). Information was collected on which PRO data are important to collect, on what frequency, and how to capture PWH’s experience on GT.

    Results: The focus groups emphasized the need for a simple tool. The PRO data identified to collect were bleeds, treatments, and health related quality of life (HR-QOL) including EQ-5D-5L, the Patient Reported Outcomes Burdens and Experiences (PROBE) and coreHEM (mental health questionnaire). It was determined that a standard mobile phone application is not the ideal method of collecting data in the event of infrequent events. Instead, a simple prompting system will be used. All PWH will be prompted via a text message or email to answer 2 questions and requests them to complete one HR-QOL questionnaire, on a rotational basis.

    Conclusion(s): myGTR allows PWH who receive GT to continue providing data on their health status and HR-QOL in a simple manner. Both GTR and myGTR enable PWH who receive GT to actively contribute to our understanding of risks and benefits of gene therapy and the success of GT in the long run.