Bleed Treatment with Eptacog Beta (rFVIIa) Results in a Low Incidence of Rebleeding in Adult and Adolescent Patients with Haemophilia A or B with Inhibitors

Authors: Dunn, A; Dargaud, Y; Abajas, Y; Carcao, M; Castaman, G; Giermasz, A; Hermans, C; Jimenez-Yuste, V; Lewandowska, M; Mahlangu, J; Meeks, S; Miesbach, W; Recht, M; Salinas, V; Chrisentery-Singleton, T; Bonzo, D; Mitchell, IS; Wilkinson, TA; Young, G

Affiliations: Nationwide Childrens Hosp, Columbus, OH 43210 USA; Ohio State Univ, Coll Med, Columbus, OH 43210 USA; Univ Lyon, Lyon, France; Univ North Carolina Chapel Hill, Hemophilia & Thrombosis Ctr, Chapel Hill, NC USA; Hosp Sick Children, Toronto, ON, Canada; Careggi Univ Hosp, Ctr Bleeding Disorders & Coagulat, Florence, Italy; Univ Calif Sacramento, Sacramento, CA USA; Catholic Univ Louvain, Clin St Luc, Brussels, Belgium; Hosp Univ La Paz, Madrid, Spain; Indiana Hemophilia & Thrombosis Ctr, Indianapolis, IN USA; Univ Witwatersrand, Natl Hlth Lab Serv, Johannesburg, South Africa; Natl Hlth Lab Serv, Johannesburg, South Africa; Goethe Univ Hosp, Frankfurt, Germany; Yale Univ, Pediat Hematol Oncol, Sch Med, New Haven, CT USA; Ctr Inherited Blood Disorders, Orange, CA USA; Amer Thrombosis & Hemostasis Network, Rochester, NY USA; LFB USA Inc, Framingham, MA USA; HEMA Biol LLC, Louisville, KY USA; GLOVAL LLC, Broomfield, CO USA; Childrens Hosp Los Angeles, Los Angeles, CA USA; Univ Southern Calif, Keck Sch Med, Los Angeles, CA USA ;

Publication: Haemophilia. 2025; 31. 78–86

ABSTRACT: INTRODUCTION Eptacog beta is a novel human recombinant FVIIa approved for use in the United States, European Union, United Kingdom and Mexico for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors (>= 12 years). It is also indicated for perioperative care in the same patient population in Europe and the United Kingdom. AIM To assess the incidence of rebleeding and review treatment outcomes in subjects with haemophilia with inhibitors enrolled in the phase 3 PERSEPT 1 clinical trial. METHODS To treat mild/moderate bleeding episodes (BEs), subjects administered an initial 75 or 225 mu g/kg dose of eptacog beta, followed (if necessary) by additional 75 mu g/kg doses at predefined intervals until bleed control. This analysis used subject-reported rebleeding to determine a rebleeding incidence for the first 24 h. Rebleeding through later timepoints was an exploratory, intention-to-treat analysis of bleed treatment data. RESULTS Four hundred and sixty-five BEs were analyzed. Through 24 h, the proportion of rebleeds was 0% (initial 75 mu g/kg dose) and 0.5% (initial 225 mu g/kg dose). Through 48 h, the proportion of rebleeds was 3.2% (75 mu g/kg initial dose) and 5.6% (225 mu g/kg initial dose); the difference between initial dose strategies was not statistically significant. The majority of rebleeds were controlled with a single dose of eptacog beta and no subject who treated a rebleed required hospitalization. CONCLUSION Subjects with haemophilia with inhibitors who used eptacog beta to treat mild/moderate BEs experienced a low incidence of rebleeding. Rebleeds that did occur were effectively controlled with eptacog beta (median, one dose) without the need for hospitalization.