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

Miguel Escobar, James Luck, Yevhenii Averianov, Jonathan Ducore, Maria Fernanda López Fernández, Adam Giermasz, Daniel P. Hart, Janna Journeycake, Craig Kessler, Cindy Leissinger, Johnny Mahlangu, Laura Villarreal Martinez, Wolfgang Miesbach, Ismail Haroon Mitha, Doris Quon, Mark T. Reding, Jean François Schved, Oleksandra Stasyshyn, Kateryna V. Vilchevska, Michael WangJerzy Windyga, W. Allan Alexander, Ahmad Al-Sabbagh, Daniel Bonzo, Ian S. Mitchell, Thomas A. Wilkinson, Cédric Hermans

Research output: Contribution to journalArticlepeer-review

Abstract

Introduction: Surgical procedures in persons with haemophilia A or B with inhibitors (PwHABI) require the use of bypassing agents (BPA) and carry a high risk of complications. Historically, only two BPAs have been available; these are reported to have variable responses. Aim: To prospectively evaluate the efficacy and safety of a new bypassing agent, human recombinant factor VIIa (eptacog beta) in elective surgical procedures in PwHABI in a phase 3 clinical trial, PERSEPT 3. Methods: Subjects were administered 200 µg/kg (major procedures) or 75 µg/kg eptacog beta (minor procedures) immediately prior to the initial surgical incision; subsequent 75 µg/kg doses were administered to achieve postoperative haemostasis and wound healing. Efficacy was assessed on a 4-point haemostatic scale during the intra- and postoperative periods. Anti-drug antibodies, thrombotic events and changes in clinical/laboratory parameters were monitored throughout the perioperative period. Results: Twelve subjects underwent six major and six minor procedures. The primary efficacy endpoint success proportion was 100% (95% CI: 47.8%–100%) for minor procedures and 66.7% (95% CI: 22.3%–95.7%) for major procedures; 81.8% (95% CI: 48.2%–97.7%) of the procedures were considered successful using eptacog beta. There was one death due to bleeding from a nonsurgical site; this was assessed as unlikely related to eptacog beta. No thrombotic events or anti-eptacog beta antibodies were reported. Conclusion: Two eptacog beta dosing regimens in PwHABI undergoing major and minor surgical procedures were well-tolerated, and the majority of procedures were successful based on surgeon/investigator assessments. Eptacog beta offers clinicians a new potential therapeutic option for procedures in PwHABI.

Original languageEnglish (US)
JournalHaemophilia
DOIs
StateAccepted/In press - 2021

Keywords

  • eptacog beta
  • haemophilia
  • inhibitors
  • PERSEPT
  • recombinant FVIIa
  • SEVENFACT
  • surgery

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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