Efficacy and Safety of Eptacog Beta (Recombinant Activated FVII) in Patients with Hemophilia A or B with Inhibitors According to Time to Initial Infusion: A Post-Hoc Analysis from PERSEPT-1
Recommended Citation
Hermans C, Escobar M, Abajas Y, Acharya S, Alvarez-Román M, Batsuli G, Dargaud Y, Janbain M, Kuriakose P, Mahlangu J, Mancuso M, Miesbach W, Yuan S, Mitchell I, Carcao M. Efficacy and Safety of Eptacog Beta (Recombinant Activated FVII) in Patients with Hemophilia A or B with Inhibitors According to Time to Initial Infusion: A Post-Hoc Analysis from PERSEPT-1. Res Pract Thromb Haemost 2023; 7.
Document Type
Conference Proceeding
Publication Date
10-1-2023
Publication Title
Res Pract Thromb Haemost
Abstract
Background: Early resolution of bleeding episodes (BEs) may reduce BE-related complications for patients with hemophilia A/B with inhibitors (PwHABI), including pain, musculoskeletal complications, absenteeism from work/school and hospitalization. The impact of time to initial dose of rFVIIa on bleed resolution is poorly understood/documented. Aims:We evaluated the impact of time to initial infusion and dosing regimen on bleed resolution and safety of eptacog beta. Methods: PERSEPT-1 was a Phase 3 trial of eptacog beta for the treatment of BEs in PwHABI (NCT02020369). Patients received an initial eptacog beta dose regimen (IDR) of either 75 or 225 μg/kg, followed by 75 μg/kg at predefined intervals over 24 hours until hemostasis was achieved. Primary endpoint was the proportion of successfully treated BEs (i.e. ‘good’/ ’excellent’ response) 12 hours after initial infusion. Safety assessments included physical examination, laboratory and immunogenicity testing, and assessment of adverse events (AEs). This post-hoc analysis evaluated the efficacy and safety of eptacog beta according to time between start of bleed and first infusion (<30 (N = 228), 30–60 (N = 105) and ≥60 (N = 73) min). Results: Across 468 BEs treated, the overall success proportion for the primary endpoint was 85.3% (95% CI 77.0–93.5) with 81.0% (70.9–91.0) and 90.3% (82.9–97.7) in the 75 and 225 μg/kg IDRs, respectively. Initial infusion at < 30 min was associated with numerically higher success proportion for both IDRs, compared with an initial infusion at 30–60 and ≥60 min; but the differences did not reach statistical significance. No significant differences were found when comparing both IDRs (Figure 1). Seven treatment-related AEs occurred in 2 patients, independently of the time between bleed start and first infusion. There were no thromboembolic events, hypersensitivity reactions, or deaths (Table 1). No neutralizing antibodies to eptacog beta were detected. Conclusion(s): Eptacog beta resulted in successful resolution of BEs with both IDRs with no difference in safety, regardless of the time to initial infusion. [Figure presented] [Table presented]
Volume
7
