Abstract Number: PB1188
Meeting: ISTH 2022 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Management of Bleeding and Trauma
Background: Prophylaxis with non-factor replacement therapy has significantly reduced the incidence of spontaneous bleeds in patients with hemophilia. However, bypassing agents remain the first line to treat traumatic bleeding episodes (BEs). Eptacog beta is a new bypassing agent approved in the US for BE treatment in patients with hemophilia A or B with inhibitors (PwHABI) aged ≥12 years.
Aims: To evaluate eptacog beta efficacy and safety for treatment of spontaneous versus traumatic BEs in PwHABI.
Methods: PERSEPT 1 was a randomized, cross-over, phase 3 trial (NCT02020369, LFB funded) in which PwHABI aged ≥12-75 years received eptacog beta for treatment of BEs using an initial dose regimen (IDR) of either 75 or 225μg/kg followed by 75μg/kg at predefined intervals over 24 hours. The primary endpoint, per EMA definition, was the proportion of successfully treated BEs (i.e., ‘good’/‘excellent’ response), irrespective of BE severity, 12 hours after the initial dose. IEC/IRB approval was granted, and all patients (or parents/legal guardians) provided informed consent.
Results: Among the 27 patients in PERSEPT 1, 27 had spontaneous BEs (197 BEs treated with 75µg/kg IDR and 184 BEs treated with 225µg/kg IDR) and 21 had traumatic BEs (53 BEs treated with 75µg/kg IDR and 32 BEs treated with 225µg/kg IDR). The location of BEs is presented in Figure 1. A similarly high proportion of spontaneous and traumatic BEs achieved successful resolution at 12 and 24 hours (Table 1). Two patients experienced seven treatment-related adverse events [infusion site discomfort (n=4), infusion site hematoma (n=2) and body temperature increase (n=1)] during 2 spontaneous and 3 traumatic BEs. There were no thromboembolic events, hypersensitivity reactions, or deaths; no neutralizing antibodies to eptacog beta were detected.
Conclusion(s): Both eptacog beta IDRs achieved successful and safe resolution of spontaneous and traumatic BEs at 12 and 24 hours in adolescent and adult PwHABI.
To cite this abstract in AMA style:
Windyga J, Meeks S, Acharya S, Boggio L, Bonanad S, Castaman G, Harroche A, Journeycake J, Mahlangu J, Malec L, McGuinn C, Nowak-Göttl U, Rafique A, Reding M, Macie C, Wang H, Hermans C. Efficacy and safety of eptacog beta for resolution of spontaneous versus traumatic bleeding episodes in patients with hemophilia A or B with inhibitors [abstract]. https://abstracts.isth.org/abstract/efficacy-and-safety-of-eptacog-beta-for-resolution-of-spontaneous-versus-traumatic-bleeding-episodes-in-patients-with-hemophilia-a-or-b-with-inhibitors/. Accessed March 22, 2024.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-and-safety-of-eptacog-beta-for-resolution-of-spontaneous-versus-traumatic-bleeding-episodes-in-patients-with-hemophilia-a-or-b-with-inhibitors/