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Conference Proceeding

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Introduction: Eptacog beta (CEVENFACTA®) is a new rFVIIa approved by the EMA for the treatment of bleeding events and prevention of bleeding during surgery in persons with haemophilia A/B with inhibitors (PwHABI) aged ≥12 years (y).

Methods: PERSEPT 3 was a Phase 3 (NCT02020369) trial of eptacog beta in PwHABI who required surgical procedures. Eptacog beta was administered at an initial dose of 200μg/kg or 75μg/kg for major or minor procedures respectively. This was followed by 75μg/kg for ≥5 (major procedures) or ≥2 (minor procedures) days. Haemostatic efficacy was assessed using a 4-point scale during the intra and postoperative care period (primary efficacy endpoint was determined by the investigator at the study centre 48±4h after the last dose of eptacog beta, based on the totality of the assessments performed on the patient (pt) at each timepoint). This post-hoc analysis compared the efficacy and safety of eptacog beta by age (pts aged <12 vs ≥12y).

Results: Twelve pts were included (<12y: n=5, 1 major and 4 minor procedures; ≥12y: n=7, 5 major and 2 minor procedures). The primary endpoint success proportion was 100% (95% CI: 39.8-100) in pts aged <12y (4 successes, 1 missing) and 71.4% (95% CI: 29.0-96.3) in pts aged ≥12y (5 successes; 2 failures). The intraoperative success proportion was 100% (95% CI: 47.8-100) for pts aged <12y (5 successes) and 100% (95% CI: 59.0-100) for pts aged ≥12y (7 successes). The success proportion 24h post-procedure was 100% (95% CI: 47.8-100) for pts aged <12y (5 successes) and 100% (95% CI: 47.8-100) for pts aged ≥12y (5 successes; 2 missing). Two pts discontinued treatment (1 aged <12y withdrew consent; 1 aged ≥12y due to an adverse event (AE): postprocedural hematoma). One pt experienced 2 serious AEs leading to death, both were considered unrelated to the treatment. No allergic or thrombotic events occurred; no neutralising antibodies were detected. Antifibrinolytics were used concomitantly with eptacog beta in 4 patients without any safety concerns.

Discussion/Conclusion: This post-hoc subgroup analysis shows that eptacog beta is effective and well-tolerated in perioperative care irrespective of patient age (<12 vs ≥12y), supporting the use of eptacog beta for bleed management (prevention and treatment) in major and minor surgical procedures in all PwHABI.







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