Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P®)

Arthur R. Thompson, J. C. Gill, B. M. Ewenstein, G. Mueller-Velten, B. A. Schwartz, T. Abshire, R. Ambinder, A. Angiolillo, Y. Arkel, D. Beardsley, R. Benjamin, M. Binder, S. Bobrow, F. Charbel, B. Cooper, N. Cornell, E. Czapek, P. Eisenberg, B. Ewenstein, S. Garcia-OrtizD. Gastineau, J. Gill, D. Green, G. Heggie, M. Heisel, H. Joist, C. Knupp, B. Lewis, D. Matthews, P. McKenna, S. Murukutla, J. Palascak, J. Penner, Jerry S Powell, H. Ritter, R. Francisco, A. Shapiro, B. Silver, T. Simpson, M. Stanek, A. R. Thompson, J. Tongol, L. Waterbury, G. White, R. Young

Research output: Contribution to journalArticle

82 Scopus citations

Abstract

von Willebrand disease (VWD) is characterized by insufficient von Willebrand factor (VWF) activity. It has been proposed that VWF:ristocetin cofactor (VWF:RCo) activity may be useful in evaluating the response to VWD treatment in patients who require replacement therapy. This prospective, open-label, non-randomized study evaluated the safety and efficacy of a factor VIII (FVIII)/VWF concentrate (Humate-P®) used in treatment regimens based on VWF:RCo activity in subjects with VWD in situations requiring urgent and necessary surgery. This article summarizes the results for 39 subjects with 42 evaluable surgical treatment events, 100% of which were rated as excellent/good for overall efficacy (achievement of haemostasis). The median loading dose based upon VWF:RCo activity was 82.3 international units/kilogram (IU kg-1; range 32.5-216.8 IU kg-1), and the median maintenance dose per infusion was 52.8 IU kg-1 (range 24.2-196.5 IU kg-1) for a median of 3 days (range 1-50 days). The median number of infusions per event was 6 (range 1-67 infusions). Three unanticipated adverse events (peripheral oedema, extremity pain and pseudo-thrombocytopenia) from two surgical treatment events were reported that were potentially treatment-related. No serious drug-related adverse events (AEs) were observed, and no thrombotic events were reported in this study. This study supports the safety and efficacy of the FVIII/VWF concentrate Humate-P® for the prevention of surgical haemorrhage in patients with VWD when administered in doses calculated in VWF:RCo units.

Original languageEnglish (US)
Pages (from-to)42-51
Number of pages10
JournalHaemophilia
Volume10
Issue number1
StatePublished - Jan 2004
Externally publishedYes

Keywords

  • Cofactor units
  • Factor III
  • Haemorrhage
  • Humate-P®
  • Ristocetin
  • Surgery
  • Von Willebrand disease

ASJC Scopus subject areas

  • Hematology

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    Thompson, A. R., Gill, J. C., Ewenstein, B. M., Mueller-Velten, G., Schwartz, B. A., Abshire, T., Ambinder, R., Angiolillo, A., Arkel, Y., Beardsley, D., Benjamin, R., Binder, M., Bobrow, S., Charbel, F., Cooper, B., Cornell, N., Czapek, E., Eisenberg, P., Ewenstein, B., ... Young, R. (2004). Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P®). Haemophilia, 10(1), 42-51.