Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94–9027 in severe haemophilia A: final results of the PROTECT VIII extension study

Mark T. Reding, Ingrid Pabinger, Pal Andre Holme, Lone Poulsen, Claude Negrier, Pavani Chalasani, Monika Maas Enriquez, Maria Wang, Karina Meijer, Maria Elisa Mancuso, Shadan Lalezari

Research output: Contribution to journalArticlepeer-review

Abstract

Introduction: The phase 2/3 PROTECT VIII main study demonstrated efficacy and safety of BAY 94–9027 (damoctocog alfa pegol; Jivi®), a B-domain-deleted recombinant factor VIII (FVIII), site-specifically PEGylated to extend its half-life. Aim: To report the final efficacy and safety data for BAY 94–9027 from the PROTECT VIII extension. Methods: Previously treated males aged 12–65 years with severe haemophilia A (FVIII <1%) who completed the multicentre, open-label PROTECT VIII main study were eligible for the extension. Patients received either on demand or prophylaxis treatments (30‒40 IU/kg twice weekly [2 × W], 45‒60 IU/kg every 5 days [E5D], or 60 IU/kg every 7 days [E7D]) and could switch to any prophylaxis regimen (variable frequency) as needed. Annualised bleeding rates (ABR), zero bleeds and safety outcomes were included in this final analysis. Results: At extension completion, patients (n = 121) received BAY 94–9027 for a median (range) total time of 3.9 (0.8–7.0) years. Median (Q1; Q3) total ABR was 1.49 (0.36; 4.80) for prophylaxis patients (n = 107), compared with 34.09 (20.3; 36.6) for on-demand patients (n = 14). Median total ABRs for 2 × W (n = 23), E5D (n = 33), E7D (n = 23) and variable frequency (n = 28) groups were 1.57, 1.17, 0.65 and 3.10, respectively. Of prophylaxis patients, 20.6% were bleed-free during the entire extension (median time, 3.2 years) and 50.0% were bleed-free during the last 6 months. No patient developed FVIII inhibitors. No deaths or thrombotic events were reported. Conclusions: Efficacy and safety of BAY 94–9027 was confirmed, with extension data supporting its use as a long-term treatment option for patients with haemophilia A.

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

Bibliographical note

Publisher Copyright:
© 2021 John Wiley & Sons Ltd

Keywords

  • Extended half-life
  • haemophilia A
  • PEGylated
  • prophylaxis
  • recombinant proteins

PubMed: MeSH publication types

  • Journal Article

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