Targeting von Willebrand disease: the current status and future directions of management therapies

von Willebrand disease (VWD) is the commonest inherited bleeding disorder, and is typically caused by deficits in the quantity or quality of von Willebrand factor (VWF).

This review describes the main clinical, diagnostic, and therapeutic aspects of VWD, with particular attention to its management. In addition, standard and avant-garde replacement therapies based on the use of VWF are discussed.

The goal of treatment for VWD is to reverse the double hemostatic defect resulting from the abnormal or reduced expression of VWF and the concomitant factor VIII (FVIII) deficiency. Treatment consists of managing any bleeds and both short-term prophylaxis (i.e. for surgery or invasive procedures) and long-term prophylaxis. While desmopressin is suitable for most patients with type 1 VWD, VWF/FVIII concentrates are the treatment of choice for the other types of VWD. Beside plasma-derived VWF/FVIII concentrates, whose safety and efficacy have been demonstrated by several clinical trials, products containing only VWF, obtained by plasma fractionation and recombinant DNA technology, have become available and marketed more recently. The clinical use of these VWF-only products is particularly attractive in the setting of surgery and long-term prophylaxis, such as the prevention of recurrent gastrointestinal bleeding in cases of angiodysplasia.