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Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A

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Author

  • MD Tarantino
  • PW Collins
  • CRM Hay
  • AD Shapiro
  • RA Gruppo
  • Erik Berntorp
  • GL Bray
  • SA Tonetta
  • PC Schroth
  • AD Retzios
  • SS Rogy
  • MG Sensel
  • BM Ewenstein
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Summary, in English

The efficacy and safety of an advanced category recombinant antihaemophilic factor produced by a plasma- and albumin-free method (rAHF-PFM) was studied in 111 previously treated subjects with haemophilia A. The study comprised a randomized, double-blinded, crossover pharmacokinetic comparison of rAHF-PFM and RECOMBINATE rAHF (R-FVIII); prophylaxis (three to four times per week with 25-40 IU kg(-1) rAHF-PFM) for at least 75 exposure days; and treatment of episodic haemorrhagic events. Median age was 18 years, 96% of subjects had baseline factor VIII <1%, and 108 received study drug. Bioequivalence, based on area under the plasma concentration vs. time curve and adjusted in vivo recovery, was demonstrated for rAHF-PFM and R-FVIII. Mean (+/-SD) half-life for rAHF-PFM was 12.0 +/- 4.3 h. Among 510 bleeding events, 473 (93%) were managed with one or two infusions of rAHF-PFM and 439 (86%) had efficacy ratings of excellent or good. Subjects who were less adherent to the prophylactic regimen had a higher bleeding rate (9.9 episodes subject(-1) year(-1)) than subjects who were more adherent (4.4 episodes subject(-1) year(-1); P < 0.03). One subject developed a low titre, non-persistent inhibitor (2.0 BU) after 26 exposure days. These data demonstrate that rAHF-PFM is bioequivalent to R-FVIII, and suggest that rAHF-PFM is efficacious and safe, without increased immunogenicity, for the treatment of haemophilia A.

Publishing year

2004

Language

English

Pages

428-437

Publication/Series

Haemophilia

Volume

10

Issue

5

Document type

Journal article

Publisher

Wiley-Blackwell

Topic

  • Hematology

Keywords

  • plasma-free method
  • haemophilia A
  • recombinant factor VIII
  • recombinant antihaemophilic factor
  • factor VIII

Status

Published

Research group

  • Clinical Coagulation, Malmö

ISBN/ISSN/Other

  • ISSN: 1351-8216