Eptacog beta is a well-tolerated and effective bypassing agent (BPA) for major and minor surgical procedures in pediatric and adult patients with hemophilia A or B with inhibitors (PwHABI), according to results from the phase 3 PRESEPT trial published in Hemophilia.
The BPA human recombinant factor VIIa (eptacog beta) was prospectively evaluated in 12 individuals, 6 undergoing major orthopedic procedures of the lower extremities and 6 undergoing minor surgical procedures. Patients were treated preoperatively with 200 μg/kg for major procedures or 75 μg/kg for minor procedures before the initial surgical incision.
Additional doses of 75 μg/kg to achieve postoperative hemostasis and wound healing were provided for at least 5 days for major procedures, or 2 days for minor procedures. Antifibrinolytics could be used.
Response to treatment was assessed by the physician using a 4-point hemostasis evaluation scale, and intraoperative success was 100%. Success proportion was 100% for minor procedures and 66.7% for major procedures at both 24- and 48-hours after the procedures. Bleeding complications occurred in 2 major orthopedic procedures that were not resolved with the use of eptacog beta and resulted in study discontinuation.
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There were no thrombotic, hypersensitive, or anaphylactic events reported. Eptacog beta was not immunogenic and none of the participants tested positive for anti-eptacog beta antibodies. Reported adverse events were deemed to not be related to concomitant eptacog beta/antifibrinolytic use.
The authors noted that “both hip and knee arthroplasty have a historically high incidence of bleeding complications in inhibitor patients, and it can be common to see delayed postoperative hemorrhage that requires treatment with alternative or alternating hemostatic agents, readmission, and/or an extended treatment period.”
This is why the guidelines state that a multidisciplinary team must be involved, a plan should be created that includes risk mitigation strategies, and aggressive and lengthy postoperative use of BPAs should be utilized.
Based on the results of this study, eptacog beta may be considered a new hemostatic agent for perioperative care in patients with hemophilia A or B with inhibitors.
Escobar M, Luck J, Averianov Y, et al. PERSEPT 3: a phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors. Haemophilia. 2021;27:911-920. doi:10.1111/hae.14418