Von Willebrand factor/factor VIII concentrates in the treatment of von Willebrand disease

Blood Coagul Fibrinolysis. 2009 Mar;20(2):89-100. doi: 10.1097/MBC.0b013e3283254570.

Abstract

Therapy for von Willebrand disease (VWD) aims to restore the hemostatic function conferred by von Willebrand factor (VWF), which facilitates platelet adhesion and aggregation, and serves to increase potentially low coagulation factor VIII (FVIII) in plasma. In patients unresponsive to desmopressin (DDAVP), the preferred treatment is with plasma-derived VWF-containing FVIII concentrates. Only a few of the available VWF/FVIII concentrates have been licensed for use in VWD based on prospective studies. The efficacy of VWF/FVIII concentrates depends on the content and quality of VWF and FVIII. Several studies have demonstrated the variability of the VWF contents, as well as the differences in the VWF multimer patterns (including the high molecular weight VWF multimers that are most effective in restoring hemostasis), among these concentrates. Treating physicians should be aware of these disparities and the potential clinical implications for patients with different VWD subtypes. Dosing has traditionally been calculated based on the FVIII content of the products, although dosing based on VWF functional activity [e.g., VWF ristocetin cofactor activity (VWF:RCo)] addresses the primary protein deficiency in VWD patients. Several clinical studies have demonstrated the efficacy of concentrates dosed according to VWF:RCo. Dosing is generally consistent across VWD subtypes, although patients with severe phenotypes or undergoing major procedures may require more infusions or longer treatment duration. Other considerations for the use of VWF-containing concentrates include laboratory monitoring of efficacy and safety issues such as thrombosis risk and thromboprophylaxis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Deamino Arginine Vasopressin / therapeutic use
  • Drug Combinations
  • Factor VIII / adverse effects
  • Factor VIII / analysis
  • Factor VIII / therapeutic use*
  • Hemostasis / drug effects
  • Hemostatics / therapeutic use
  • Humans
  • Platelet Adhesiveness / drug effects
  • Platelet Aggregation / drug effects
  • Thrombosis / blood
  • Thrombosis / chemically induced
  • Thrombosis / prevention & control
  • Time Factors
  • von Willebrand Diseases / blood
  • von Willebrand Diseases / drug therapy*
  • von Willebrand Factor / adverse effects
  • von Willebrand Factor / analysis
  • von Willebrand Factor / therapeutic use*

Substances

  • Drug Combinations
  • Hemostatics
  • factor VIII, von Willebrand factor drug combination
  • von Willebrand Factor
  • Factor VIII
  • Deamino Arginine Vasopressin