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)
Pages (from-to)911-920
Number of pages10
JournalHaemophilia
Volume27
Issue number6
DOIs
StatePublished - Nov 2021

Bibliographical note

Funding Information:
The Program for the Evaluation of Recombinant Factor Seven Efficacy by Prospective Clinical Trial - PERSEPT was funded by LFB SA. The authors would like to thank the participants of the PERSEPT 3 trial and their families, without whom none of this work would be possible. The authors also wish to thank the other investigators at each study site and the Data Monitoring Committee members: Drs. Santiago Bonanad Boix (Spain); Vasily Mamonov (Russia); Philippe de Moerloose (Switzerland); and Alok Srivastava (India), as well as Sonia Nasr, PhD for her assistance during the editing of this manuscript.

Funding Information:
The Program for the Evaluation of Recombinant Factor Seven Efficacy by Prospective Clinical Trial ‐ PERSEPT was funded by LFB SA. The authors would like to thank the participants of the PERSEPT 3 trial and their families, without whom none of this work would be possible. The authors also wish to thank the other investigators at each study site and the Data Monitoring Committee members: Drs. Santiago Bonanad Boix (Spain); Vasily Mamonov (Russia); Philippe de Moerloose (Switzerland); and Alok Srivastava (India), as well as Sonia Nasr, PhD for her assistance during the editing of this manuscript.

Publisher Copyright:
© 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.

Keywords

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

PubMed: MeSH publication types

  • Clinical Trial, Phase III
  • Journal Article

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