Abstract Number: PB1174
Meeting: ISTH 2022 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Management of Bleeding and Trauma
Background: Eptacog beta (LFB/HEMA Biologics) is a new bypassing agent approved in the US for treatment of bleeding episodes (BEs) in patients with hemophilia A/B with inhibitors (PwHABI) aged ≥12 years.
Aims: To evaluate eptacog beta for treatment of BEs according to low ( < 5 BU) or high (≥5 BU) inhibitor titer.
Methods: This analysis pertains to PERSEPT 1, a phase 3 study performed by LFB in PwHABI aged ≥12 years (NCT02020369). IEC/IRB approvals and informed consent were obtained. PwHABI received eptacog beta for treatment of BEs at an initial dose of 75 or 225μg/kg, followed by 75μg/kg at predefined intervals over 24 hours. The primary endpoint (EMA definition) was the proportion of successfully treated BEs (i.e., ‘good’/‘excellent’ response), irrespective of BE severity, 12 hours after initial dose.
Results: Twenty-seven patients with 468 BEs were analyzed (low-titer subgroup, 218 BEs; high-titer subgroup, 250 BEs) (Table 1). The proportion (95%CI) of successfully treated BEs at 12 hours with 75 and 225µg/kg initial dose regimens (IDRs), respectively, was 75.2% (63.6–86.8) versus 87.1% (79.8–94.5) in the low-titer subgroup (p=0.006), and 94.6% (88.2–100) versus 100% (100–100) in the high-titer subgroup (p=0.034) (Figure 1). Corresponding results at 24 hours were 96.4% (91.5–100) versus 99.0% (97.1–100) in the low-titer subgroup (p=0.118), and 96.9% (92.4–100) versus 100% (100–100) in the high-titer subgroup (p=0.125). Two patients in the low-titer subgroup experienced seven treatment-related adverse events (infusion site discomfort [n=4]; infusion site hematoma [n=2]; body temperature increase [n=1]). None occurred in the high-titer subgroup. No thromboembolic events, hypersensitivity reactions, or deaths were reported. No eptacog beta neutralizing antibodies were detected.
Conclusion(s): Both eptacog beta IDRs achieved successful resolution of BEs in PwHABI irrespective of inhibitor titer with a higher response rate for 225μg/kg IDR at 12 hours. No safety issues were noted.
To cite this abstract in AMA style:
Hart D, Sidonio R, Abajas Y, Ahuja S, Alvarez-Román M, Carcao M, Dargaud Y, Escobar M, Kessler C, Khan O, Leissinger C, Maes P, Mancuso M, Majluf Cruz A, Miesbach W, Simpson M, Wang M, Macie C, Wang H, Srivastava A. Eptacog beta for resolution of bleeding episodes in patients with hemophilia A or B with inhibitors: Efficacy and safety according to inhibitor titer [abstract]. https://abstracts.isth.org/abstract/eptacog-beta-for-resolution-of-bleeding-episodes-in-patients-with-hemophilia-a-or-b-with-inhibitors-efficacy-and-safety-according-to-inhibitor-titer/. Accessed March 21, 2024.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/eptacog-beta-for-resolution-of-bleeding-episodes-in-patients-with-hemophilia-a-or-b-with-inhibitors-efficacy-and-safety-according-to-inhibitor-titer/