Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of PERSEPT 1 & 2

• Seven of 8 life- or limb-threatening bleeds in hemophilia patients with inhibitors were controlled with eptacog beta in clinical trials.

• Eptacog beta was well tolerated, and bleed control was achieved in timeframes consistent with previous reports for similar injuries.

Severe bleeding episodes (BEs) in persons with hemophilia A or B and inhibitors (PwHABI) 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 PwHABI 12 years of age or older. The aim of this study is to assess eptacog beta efficacy and safety for severe bleed treatment in PwHABI during two phase 3 trials (PERSEPT 1 and PERSEPT 2). Subjects could treat severe BEs with initial doses of 75 or 225 μg/kg eptacog beta at home, with subsequent 75 μg/kg eptacog beta infusions taking place 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 subject was assessed by computed tomography. Seven PwHABI (ages 1-50 y) 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 min to 96 h. 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. NCT02020369. NCT02448680