Therapy of von Willebrand disease.

Abstract

Impressive progress has been made in the treatment of von Willebrand disease. Desmopressin is the drug of choice for mild to moderate type I von Willebrand disease patients in whom adequate hemostatic levels of vWF activity can be achieved. Cryoprecipitate from random donors is no longer an optimal choice because of the risk of transfusion-associated viral infections. In special circumstances, cryoprecipitate from a very small donor pool, particularly if obtained following desmopressin stimulation, remains an attractive alternative because these donors can be intensively screened. This therapy may also be somewhat less expensive than commercial concentrates. Virally attenuated commercial concentrates containing intact vWF multimers are currently the treatment of choice to achieve high levels of vWF for moderate to severe von Willebrand disease and for patients with variants of von Willebrand disease that cannot be adequately treated with desmopressin or for whom desmopressin is contraindicated. It is hoped that concentrates of vWF specifically designed for treatment of von Willebrand disease will prove to be safe and efficacious. Standardized assays of vWF in concentrates need to be established. Although, the optimal treatment product has not been produced, several of the more recently developed products have structures that more closely resemble intact normal plasma vWF and appear promising.