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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0225 - High Bleed Control with a Single 225 µg/kg Eptacog Beta Dose in Adult and Adolescent Hemophilia A or B Persons with Inhibitors

Sunday, June 23, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall

    Presenting Author(s)

  • Miguel Escobar, MD

    Professor of Medicine and Pediatrics
    University of Texas Health Science Center of Houston, McGovern Medical School, Gulf States Hemophilia and Thrombophilia Center, Houston, Texas
    Houston, Texas, United States

    • Co-Author(s)

  • Wolfgang Miesbach, MD

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

  • SB

    Santiago Bonanad

    Head, Haemophilia Unit, Thrombosis and Haemostasis Unit
    University and Polytechnic Hospital La Fe
    Valencia, Comunidad Valenciana, Spain

  • Manuel Carcao

    Professor
    Department of Pediatrics, Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Canada
    Toronto, Ontario, Canada

  • Giancarlo Castaman

    Medical Director Hematologist
    Department of Oncology Careggi University Hospital, Florence, Italy
    Florence, Toscana, Italy

  • Yesim G. Dargaud, MD,PhD

    Professor of Medicine
    UR 4609 Hemostase et Thrombose, Universite Lyon 1
    Lyon, Rhone-Alpes, France

  • Cedric Hermans, MD, FRCP, PhD

    Head of the Division of Haematology, the Hemostasis and Thrombosis Unit, and the Hemophilia Center
    Saint-Luc University Hospital
    Saint-Luc, Brussels Hoofdstedelijk Gewest, Belgium

  • JJ

    Julie Jaffray

    Associate Professor
    Rady Children's Hospital-San Diego, University of California San Diego
    San Diego, California, United States

  • MJ

    Maissaa Janbain

    Associate Professor
    Department of Hematology/Oncology, Tulane School of Medicine, New Orleans, Louisiana, USA
    New Orleans, Louisiana, United States

  • Víctor Jiménez-Yuste

    Head of Hematology Service
    Hospital Universitario La Paz-IdiPaz
    Madrid, Madrid, Spain

  • OK

    Osman Khan

    Director
    Oklahoma Bleeding and Clotting Disorders Center at OU Health
    Oklahoma City, Oklahoma, United States

  • Magdalena Lewandowska, MD

    Indiana Hemophilia & Thrombosis Center
    Indianapolis, Indiana, United States

  • Johnny Mahlangu, MBBCh, MMed

    Head of Department and Principal Haematologist
    Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, and Charlotte Maxeke Johannesburg Academic Hospital
    Johannesburg, Gauteng, South Africa

  • Johannes Oldenburg, MD

    Professor, Chairman and Director
    University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine
    Bonn, Nordrhein-Westfalen, Germany

  • CP

    Charles Percy

    Consultant Haematologist
    University Hospitals Birmingham
    Birmingham, England, United Kingdom

  • MR

    Mark T. Reding

    Director
    Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center
    Minneapolis, Minnesota, United States

  • DB

    Daniel Bonzo

    Vice President, Global Biometry
    LFB-USA
    Framingham, Massachusetts, United States

  • IM

    Ian Mitchell

    Medical Science Liaison
    HEMA Biologics, LLC
    Louisville, Kentucky, United States

  • TW

    Thomas Wilkinson

    Consulting Scientist
    GLOVAL LLC
    Broomfield, Colorado, United States

  • Guy Young, MD

    Director, Hemostasis and Thrombosis Program
    Children's Hospital of Los Angeles, Los Angeles, USA
    Los Angeles, California, United States

Background: Achieving rapid bleed control with a single infusion of a bypassing agent reduces treatment burden in persons with hemophilia A or B with inhibitors (PwHABI) during acute bleed management. Eptacog beta is a rFVIIa that is approved for bleeding episode (BE) treatment in PwHABI ≥12 years using two initial dose regimens (IDRs) of 75 or 225µg/kg. Treatment success proportions during the PERSEPT1 trial (NCT02020369) were 82% for the 75µg/kg IDR and 91% for the 225µg/kg IDR at 12h (primary endpoint).

Aims: To investigate the efficacy of a single 225µg/kg eptacog beta dose when treating mild/moderate (not life- or limb-threatening) BEs in PwHABI ≥12 years during PERSEPT1.

Methods: Twenty-seven PwHABI (ages 12-54 years; none on prophylaxis) were randomized to treat BEs using either the 75 or 225µg/kg IDR, and switched to the alternate IDR every 3 months. Subjects assigned to the 225 IDR treated mild/moderate BEs with an initial 225µg/kg eptacog beta dose, followed after 9h by 75µg/kg q3h if needed (Figure 1). The efficacy of a single 225µg/kg dose was calculated as the treatment success proportion at 9h (the first defined hemostasis evaluation per protocol).

Results: Twenty-five out of 27 subjects treated 213 mild/moderate BEs using the 225µg/kg IDR, and a single-dose efficacy of 84% was observed. Single-dose efficacy with the 225µg/kg IDR for 182 joint and 31 non-joint BEs was 86.5% and 72.4%, respectively. Similar single-dose efficacy was observed for 58 target joint and 124 non-target joint BEs (84.6% and 87.3%, respectively) and for 182 spontaneous and 31 traumatic BEs (85.8% and 76.7%, respectively; Figure 2).

Conclusion(s): A single 225µg/kg eptacog beta dose provided effective bleed control for various mild/moderate BE types in the vast majority of cases, thereby reducing the treatment and disease burden in PwHABI, and narrowing the health equity gap between inhibitor and non-inhibitor patients.