Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients

Research output: Contribution to journalArticle

Abstract

Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.

Details

Authors
  • A. Borel-Derlon
  • A. B. Federici
  • V. Roussel-Robert
  • J. Goudemand
  • C. A. Lee
  • I. Scharrer
  • C. Rothschild
  • Erik Berntorp
  • C. Henriet
  • Z. Tellier
  • F. Bridey
  • P. M. Mannucci
Organisations
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Cardiac and Cardiovascular Systems

Keywords

  • von Willebrand disease, von Willebrand factor, concentrate
Original languageEnglish
Pages (from-to)1115-1124
JournalJournal of Thrombosis and Haemostasis
Volume5
Issue number6
Publication statusPublished - 2007
Publication categoryResearch
Peer-reviewedYes