Recombinant von Willebrand Factor for von Willebrand Disease

Patients with severe von Willebrand disease (VWD) have persistent, recurrent episodes of mucosal bleeding, menorrhagia, and bleeding with surgery or trauma. Treatment with plasma-derived von Willebrand factor (VWF) concentrates controls bleeding but exposes patients to potential infection by viruses and other microbes. A prior phase I trial showed that a recombinant VWF (rVWF) stabilized factor VIII (FVIII) and was not associated with thrombotic events (Blood 2013; 122:648).

To examine the pharmacokinetics and hemostatic efficacy of rVWF, an international group of investigators conducted an industry-sponsored, phase III trial involving 37 patients with severe VWD. For the treatment of bleeding episodes, the first of up to four infusions of r…