Current management of patients with severe von Willebrand disease type 3: a 2012 update.

Von Willebrand disease type 3 (VWD3) is the most severe form of this bleeding disorder due to the almost complete deficiency of von Willebrand factor (VWF). VWD3 is inherited as an autosomal recessive trait. While heterozygous carriers exhibit mild or no bleeding symptoms, most patients with VWD3, which is characterized by undetectable levels of VWF antigen (VWF:Ag) and reduced concentrations (<20 IU/dl) of factor VIII (FVIII), show severe bleeding symptoms. Although the incidence of VWD3 is rare, the condition is of considerable interest because of its severe clinical presentation, the need for replacement therapy and the risk of alloantibodies following infusion of plasma-derived VWF concentrates. This review, based on clinical experience, provides an update on the clinical, laboratory and molecular markers of VWD3 that can be useful for determining the optimal therapeutic approach in these patients.