Prophylaxis and von Willebrand's disease (vWD)

Von Willebrand disease (vWD) is an inherited bleeding disorder with a prevalence of approximately 1% in the general population. The bleeding occurs in this disorder primarily because of abnormalities in platelet adhesion, due to a primary deficiency or defect in the von Willebrand factor (vWF) often with a concomitant decrease in factor VIII (FVIII) levels. The mainstay of treatment for the majority of patients with vWD is desmopressin, which releases vWF from endothelial cells. For patients in which desmopressin is neither suitable or recommended, coagulation factor concentrates containing both vWF with FVIII are the alternative therapeutic option for controlling bleeding.Joint disease is an uncommon but disabling complication of vWD. The arthropathy results from recurrent bleeding into a joint, with the ankle and hip being the joints most often affected, particularly in patients with type 3 vWD. Without adequate treatment, persistent bleeding into joints can result in pain, joint degeneration, swelling and loss of range of motion (ROM). There is some evidence to suggest that joint bleeding and subsequent joint damage might be prevented by early prophylaxis. A soon to open clinical trial by the Von Willebrand Disease Prophylaxis Network (vWD PN) aims to further explore this treatment concept. This article addresses the prophylaxis of vWD, with a particular focus on joint disease.