Spontaneous and traumatic bleeding events were successfully resolved using the bypassing agent eptacog beta among patients with hemophilia A or B with inhibitors, according to the results of a post hoc analysis presented at the ISTH 2022 Congress.
A human factor VII bypassing agent, eptacog beta is approved for the treatment of bleeding episodes among patients aged 12 or older with hemophilia A or B with inhibitors. The aim of this study was to evaluate the effect of eptacog beta on spontaneous and traumatic bleeding episodes.
This was a post hoc analysis of the phase 3, cross-over PERSEPT 1 trial, which randomly assigned 50 patients 12 years or older with hemophilia A or B with inhibitors to eptacog beta at an initial dose of 75 or 225 μg/kg, both followed by 75 μg/kg over 24 hours. The primary endpoint was successfully treated bleeding episodes within 12 hours after the first dose.
At baseline, the median age was 31 and 92.6% of patients had hemophilia A. Most patients had severe disease, and the Bethesda unit for inhibitor status was less than 5 among 48.1% of patients and 5 or greater among 51.9%. The majority of spontaneous bleeding episodes occurred in the elbow, knee, or ankle/foot, and the most common locations for traumatic bleeding episodes was the elbow, soft tissue/muscle, and ankle/foot.
There were 381 total spontaneous bleeds that occurred among all patients, which resolved with eptacog beta within 12 hours among 85.1% and 93.8% of patients in the lower and higher initial dosing groups, respectively. This increased to 95.8% and 99.4%, respectively, within 24 hours.
There were a total of 85 bleeds among 32 patients who had a traumatic bleeding episode. There were 88.0% and 93.5% of patients who achieved resolution within 12 hours in the lower and higher initial dose groups, respectively. Within 24 hours, all bleeding episodes resolved.
Mild treatment-related adverse events (AEs) occurred in 2 patients, which included infusion site discomfort, infusion site hematoma, and body temperature increase. There were no treatment-related serious AEs and no thromboembolic events. Additionally, there were no neutralizing anti-eptacog beta antibodies detected.
The authors concluded that, “both eptacog beta initial dose regimens achieved successful resolution of spontaneous and traumatic bleeding episodes at 12 and 24 hours in adolescent and adult patients with hemophilia A or B with inhibitors.”
Disclosures: This study was supported by LFB.
Windyga J, Meeks S, Acharya S, et al. Efficacy and safety of eptacog beta for resolution of spontaneous versus traumatic bleeding episodes in patients with hemophilia A or B with inhibitors. Presented at: ISTH 2022 Congress; July 9-13, 2022. Abstract PB1188.