INSIGHT INTO ATHN 16: TREATMENT OF BLEEDING EVENTS IN PATIENTS WITH CONGENITAL HEMOPHILIA A OR B WITH INHIBITORS TO EVALUATE THE SAFETY OF COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW

Document Type

Conference Proceeding

Publication Date

12-9-2024

Publication Title

Am J Hematol

Keywords

emicizumab, recombinant blood clotting factor 7a, adverse drug reaction, bleeding, bleeding disorder, cardiovascular disease, child, conference abstract, drug therapy, exercise, female, Food and Drug Administration, hemophilia, hemophilia A, human, multicenter study, phase 4 clinical trial, prophylaxis, side effect, therapy, Torque teno virus infection, transfusion transmitted infection, treatment duration

Abstract

Background: Eptacog beta (rFVIIa-jncw, SEVENFACT) was approved by the U.S. Food and Drug Administration (FDA) in 2020 for the treatment of bleeding events (BEs) in individuals 12 years of age and older with hemophilia A or B complicated by inhibitors. The ATHN 16 study: Treatment of Bleeding Events in Patients with Congenital Hemophilia A or B with Inhibitors to Evaluate the Safety of Coagulation Factor VIIa (recombinant)-jncw was designed to collect real-world evidence on the safety of eptacog beta. Objective: To provide an evaluation of the safety of eptacog beta when used to treat BEs in persons with hemophilia A or B and inhibitors (PwHABI) with or without FDA-approved prophylactic treatment. Methods: ATHN 16 (NCT04647227) is a phase IV, United States-centric, multicenter, open-label, safety study enrolling PwHABI, aged 12 years and older, who are either on an FDA-approved prophylaxis therapy and at risk of experiencing a breakthrough BE or who are not on prophylactic treatment and may need to control a BE. Safety is evaluated according to European Hemophilia Safety Surveillance (EUHASS) criteria, including allergic or other acute events, treatment-emergent side effects, transfusion-transmitted infections, inhibitor (FVII) development, thrombosis, cardiovascular events, malignancies, neurological events, and deaths. After informed consent was obtained, demographics, bleeding disorder history, inhibitor history, baseline medical and surgical history were recorded. Each participant is provided nine (9) 75 μg/kg doses of eptacog beta supplied by the study funder. Eptacog beta was administered by the participant or by study staff at the time of a BE. The doses and duration of treatment were determined at the discretion of the treating physician and participant. BE details including timing of BE, any treatments associated with the BE (including eptacog beta) and timing of resolution of the BE, were collected as well as surgical procedures, EUHASS events, adverse events of special interest (AESIs), and serious adverse events (SAEs). Results: Between August 2021 and October 2023, 17 participants were enrolled at 19 ATHN-affiliated sites. All study participants with severe hemophilia received prophylactic emicizumab with one starting emicizumab prophylaxis during the trial. To date, 72 BEs have been treated with eptacog beta. Of these bleeds, 29 were classified as spontaneous, 31 were traumatic, 11 were after activity or exercise, and 1 was classified as other (medical procedure). No adverse events (AEs) related to eptacog beta use were reported. Two AEs, unrelated to the study product, were reported and resolved. Three SAEs, unrelated to the study product were reported and resolved. Conclusions: To date, the safety profile of eptacog beta for the treatment of BEs in the ATHN 16 participants is consistent with previously published reports.

Volume

100

First Page

24

Last Page

25

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