Treatment of von Willebrand's Disease

Early recognition that plasma infusions could correct the prolonged bleeding time and factor VIII deficiency in von Willebrand's disease (vWD) prompted major research efforts. Identification of the factor VIII molecule, its components, and its functions has provided critical information about replacement requirements to prevent or control bleeding. Plasma and cryoprecipitate have been the mainstay of therapy. Until recently, lyophilized coagulation factor concentrates had little usefulness in treating vWD. Current factor VIII concentrates prepared by monoclonal or recombinant technology (or both) are devoid of von Willebrand factor. Desmopressin has been a revolutionary synthetic agent useful in numerous patients with mild or moderately severe vWD. In many patients, however, desmopressin is ineffective. Only a few currently available factor VIII concentrates contain biologically active von Willebrand factor and can be used to prepare patients for surgical procedures or to stop hemorrhage. Inhibitors of von Willebrand factor arise de novo or in patients with vWD. Management with activated prothrombin complex concentrates or removal of antibody with protein A columns has been successful. Despite current technologic advances, not all patients with vWD can be successfully treated. Many problems with vWD continue to be unresolved.