Treatment of Severe Bleeds with Eptacog Beta in Hemophilia A or B with Inhibitors: A Post Hoc Analysis of the PERSEPT 1 and 2 Trials

Authors: Young, G; Mahlangu, J; Boggio, LN; Carcao, M; Dargaud, Y; Escobar, M; Giermasz, A; Hermans, C; Kuriakose, P; Miesbach, W; Nance, D; Rafique, A; Sidonio, RF, JR; Vilchevska, KV; Wang, M; Pipe, SW

Affiliations: Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Los Angeles, CA. Keck School of Medicine, University of Southern California, Los Angeles, CA. Department of Molecular Medicine and Haematology, Hemophilia Comprehensive Care Center, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa. Department of Pediatrics, Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL. Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada. Division of Hemostasis and Thrombosis, Lyon University Hospitals, Lyon, France. University of Texas Health Science Center at Houston, Houston, TX. Division of Hematology/Oncology, University of California Davis, Sacramento, CA. Division of Haematology, Cliniques Saint-Luc, Université Catholique de Louvain, Brussels, Belgium. Department of Internal Medicine, Henry Ford Hospital, Detroit, MI. Department of Haemostaseology, Goethe University Hospital, Frankfurt, Germany. Division of Hematology, Banner MD Anderson Cancer Center, Phoenix, AZ. Department of Pediatric Hematology and Oncology, Atrium Health Levine Cancer, Charlotte, NC. Department of Pediatrics, Emory University and Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, Atlanta, GA. Department of Pediatrics, National Specialized Children’s Hospital Okhmatdyt, Kyiv, Ukraine. Department of Pediatrics, Hemophilia and Thrombosis Center, University of Colorado, Aurora, CO. Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI.

 Publication: Blood vessels, thrombosis & hemostasis ; 2025 ; 2. 100069

ABSTRACT: Severe bleeding episodes (BEs) in persons with hemophilia A or B and inhibitors (PwHABIs) represent challenging clinical situations and can require treatment regimens lasting days or weeks before hemostatic control is achieved. Eptacog beta is a recombinant activated human factor VII bypassing agent approved for treating and controlling bleeding in PwHABIs aged ≥12 years. The aim of this study is to assess the efficacy and safety of eptacog beta for severe bleeding treatment in PwHABIs during 2 phase 3 trials (PERSEPT 1 and PERSEPT 2). Patients could treat severe BEs with initial doses of 75 or 225 μg/kg eptacog beta at home, followed by subsequent 75 μg/kg eptacog beta infusions administered at predefined intervals in a hospital or hemophilia treatment center. Satisfactory treatment responses to eptacog beta were typically defined in this post hoc analysis by physician- and patient-reported hemostasis evaluations of « excellent » or « good. » Hemostatic control of an intracranial hemorrhage (ICH) in 1 patient was assessed by computed tomography. Seven PwHABIs (aged 1-50 years) treated 8 BEs considered severe or otherwise life threatening with eptacog beta during PERSEPT 1 and PERSEPT 2. Hemostatic control of 7 of these BEs (including 3 ICH events) was achieved. Eptacog beta treatment durations ranged from 25 minutes to 96 hours. No thrombotic events were reported, and eptacog beta was well tolerated. Most severe BEs resolved with eptacog beta treatment during PERSEPT 1 and PERSEPT 2. The PERSEPT 1 and PERSEPT 2 trials were registered at www.clinicaltrials.gov as #NCT02020369 and #NCT02448680, respectively.