Preliminary Analysis of ATHN 16: Real-World Safety of Eptacog Beta
Recommended Citation
Singleton T, Acharya S, Ahuja S, Amos L, Bonzo D, Eason A, Escobar M, Knoll C, Kuriakose P, Lagrue E, Recht M, Sullivan S, Quon D, Reding M. Preliminary Analysis of ATHN 16: Real-World Safety of Eptacog Beta. Res Pract Thromb Haemost 2023; 7.
Document Type
Conference Proceeding
Publication Date
10-1-2023
Publication Title
Res Pract Thromb Haemost
Abstract
Background: Eptacog beta (rFVIIa-jncw) received regulatory approval in the U.S. in 2020 for the treatment of bleeding events (BEs) in persons with hemophilia A/B with inhibitors (PwHABI), 12 years of age and older. The ATHN 16 study was designed by the American Thrombosis and Hemostasis Network (ATHN) and the investigators to collect real-world evidence on the safety of eptacog beta. Aims: To evaluate the safety of eptacog beta when used to treat BEs in participants with HAB with inhibitors with or without prophylactic treatment. Methods: ATHN 16 (NCT04647227) is a phase IV, multi-center, open-label safety study enrolling PwHABI, who are either on long-term prophylaxis (e. g., emicizumab) or episodic treatment. ATHN 16 received central IRB approval. After informed consent is obtained, each participant is provided nine (9) 75 μg/kg doses of eptacog beta to be administered as needed for bleed treatment. BEs are treated by the participant or by study staff at the time of an event. Actual dose administered (75 mcg/kg or 225 mcg/kg) is determined at the discretion of the treating physician. The safety of eptacog beta is evaluated based on events included in the European Haemophilia Safety Surveillance (EUHASS) protocol. There are no pre-specified efficacy endpoints. Results: Between August 2021 and October 2022, eleven subjects have been enrolled, ten of whom are on prophylactic emicizumab. Twenty-eight bleeding events have been treated (57% spontaneous, 36% traumatic, 8% other (surgery, activity/exercise) in four of the eleven enrolled participants. One adverse event (AE), unrelated to study product, was reported and resolved. One serious adverse event (SAE), unrelated to study product was reported and resolved. Conclusion(s): To date, the safety profile of eptacog beta, in the ATHN 16 participants treated with eptacog beta, is favorable and consistent with previous reports. [Table presented] [Table presented]
Volume
7
