Treatment of von Willebrand Disease.

The general goal of therapy in von Willebrand disease is to correct the two defects that are related to the bleeding tendency of these patients, i.e., the abnormal primary hemostasis expressed by a prolonged bleeding time (BT) and the abnormal intrinsic coagulation due to low factor VIII (FVIII) levels. There are two main treatments in von Willebrand disease, i.e., desmopressin and transfusional therapy with blood products. Desmopressin is most effective in patients with type 1, who account for about 80 per cent of all patients. The remaining patients with type 2 and type 3 von Willebrand disease do not respond consistently to desmopressin and need the infusion of blood products containing FVIII and von Willebrand factor. Virally-inactivated concentrates that are currently on the market are preferable to fresh-frozen plasma and cryoprecipate, that still carry a small risk of transmitting blood-borne infections. In rare instance, particularly for hemorrhoages in mucosal tracts such as gastrointestinal bleeding, platelet concentrates are useful adjuncts to plasma concentrates when patients do not respond to the latters. The role of purified von Willebrand factor and of recombinant products is still unclear.