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Perioperative haemostasis with full-length, PEGylated, recombinant factor VIII with extended half-life (rurioctocog alfa pegol) in patients with haemophilia A: Final results of a multicentre, single-arm phase III trial.

Authors
  • Gruppo, Ralph1
  • López-Fernández, Maria-Fernanda2
  • Wynn, Tung T3
  • Engl, Werner4
  • Sharkhawy, Marlies4
  • Tangada, Srilatha5
  • 1 Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
  • 2 Complejo Hospitalario Universitario A Coruña, Coruña, Spain. , (Spain)
  • 3 College of Medicine, University of Florida, Gainesville, Florida.
  • 4 Baxalta Innovations GmbH, a Takeda company, Vienna, Austria. , (Austria)
  • 5 Baxalta US Inc., a Takeda company, Cambridge, Massachusetts, USA.
Type
Published Article
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
Publication Date
Sep 01, 2019
Volume
25
Issue
5
Pages
773–781
Identifiers
DOI: 10.1111/hae.13807
PMID: 31353761
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Rurioctocog alfa pegol (BAX 855, TAK-660) is a PEGylated, full-length, recombinant factor VIII (rFVIII) with extended half-life developed from unmodified rFVIII (antihaemophilic factor [recombinant]). To determine the perioperative haemostatic efficacy and safety of rurioctocog alfa pegol in male previously treated patients (PTPs) with severe haemophilia A. This multicentre, single-arm, phase III study included PTPs who were to undergo major or minor elective or minor emergency surgical, dental or other invasive procedures. Rurioctocog alfa pegol dose and frequency were individualized based on patients' pharmacokinetic profiles for major surgeries and by rurioctocog alfa pegol incremental recovery for minor surgeries. Haemostatic efficacy was assessed using the Global Haemostatic Efficacy Assessment score. Twenty-one patients aged 16-61 years underwent 21 major and five minor surgeries. For all 24 evaluable surgeries, overall haemostatic efficacy was rated as excellent and blood loss comparable to that expected in non-haemophilic patients. No blood transfusions were required intraoperatively but were administered postoperatively for four surgeries in three patients. Five injury-related postoperative bleeding episodes occurred in five patients, of which two required additional rurioctocog alfa pegol treatment. Two non-serious adverse events of mild severity (increased ALT level and headache) were considered possibly related to rurioctocog alfa pegol. There were no deaths or treatment-related serious adverse events. No patients developed inhibitory antibodies to FVIII or persistent IgG- or IgM-binding antibodies to FVIII, PEG-FVIII or PEG. Rurioctocog alfa pegol was well tolerated and effective for perioperative use in patients with haemophilia A and showed no signs of immunogenicity. © 2019 The Authors Haemophilia Published by John Wiley & Sons Ltd.

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