Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate

A prospective, multicenter study

Pier M. Mannucci, Juan Chediak, Wahid Hanna, John Byrnes, Marlies Ledford, Bruce M. Ewenstein, Anastassios D. Retzios, Barbara A. Kapelan, Richard S. Schwartz, Craig Kessler

Research output: Contribution to journalArticle

163 Citations (Scopus)

Abstract

Among patients with von Willebrand disease (VWD) who are unresponsive to desmopressin therapy, replacement with plasma-derived concentrates is the treatment of choice. Because prospective studies are lacking, such treatment has been largely empirical. A multicenter, prospective study has been conducted in 81 patients with VWD (15 patients with type 1, 34 with type 2, and 32 with type 3 disease) to investigate the efficacy of a high-purity factor VIII/von Willebrand factor (FVIII/VWF) concentrate for treatment of bleeding and surgical prophylaxis. Two preparations of the concentrate-one virally inactivated with solvent detergent, the other with an additional heat-treatment step-were evaluated. Pharmacokinetic parameters were similar for both preparations. Using pre-established dosages based on the results of pharmacokinetic studies, 53 patients were administered either preparation for the treatment of 87 bleeding episodes, and 39 patients were treated prophylactically for 71 surgical or invasive procedures. Sixty-five (74.7%) and 10 (11.5%) of the bleeding episodes were controlled with 1 or 2 infusions, respectively. Patients with severe type 3 VWD typically required more infusions and higher doses, at shorter time intervals, than did patients with generally milder types 1 and 2. Among patients undergoing surgical procedures, blood loss was lower than that predicted prospectively, and losses exceeding the predicted value did not correlate with the postinfusion skin bleeding time. In conclusion, the concentrate effectively stopped active bleeding and provided adequate hemostasis for surgical or invasive procedures, even in the absence of bleeding time correction.

Original languageEnglish (US)
Pages (from-to)450-456
Number of pages7
JournalBlood
Volume99
Issue number2
DOIs
StatePublished - Jan 15 2002

Fingerprint

von Willebrand Diseases
Multicenter Studies
Prospective Studies
Pharmacokinetics
Hemorrhage
Deamino Arginine Vasopressin
Bleeding Time
Therapeutics
Detergents
Type 3 Von Willebrand's Disease
Skin
Blood
Heat treatment
Surgical Blood Loss
Plasmas
factor VIII, von Willebrand factor drug combination
Hemostasis
Hot Temperature

All Science Journal Classification (ASJC) codes

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate : A prospective, multicenter study. / Mannucci, Pier M.; Chediak, Juan; Hanna, Wahid; Byrnes, John; Ledford, Marlies; Ewenstein, Bruce M.; Retzios, Anastassios D.; Kapelan, Barbara A.; Schwartz, Richard S.; Kessler, Craig.

In: Blood, Vol. 99, No. 2, 15.01.2002, p. 450-456.

Research output: Contribution to journalArticle

Mannucci, PM, Chediak, J, Hanna, W, Byrnes, J, Ledford, M, Ewenstein, BM, Retzios, AD, Kapelan, BA, Schwartz, RS & Kessler, C 2002, 'Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: A prospective, multicenter study', Blood, vol. 99, no. 2, pp. 450-456. https://doi.org/10.1182/blood.V99.2.450
Mannucci, Pier M. ; Chediak, Juan ; Hanna, Wahid ; Byrnes, John ; Ledford, Marlies ; Ewenstein, Bruce M. ; Retzios, Anastassios D. ; Kapelan, Barbara A. ; Schwartz, Richard S. ; Kessler, Craig. / Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate : A prospective, multicenter study. In: Blood. 2002 ; Vol. 99, No. 2. pp. 450-456.
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