Pharmacotherapy of von Willebrand disease

Introduction: Von Willebrand disease (VWD), the most common inherited bleeding disorder, is typically due to quantitative or qualitative deficiencies of von Willebrand factor (VWF).Areas covered: This review, which also summarizes the clinical, pathogenic and diagnostic features of VWD, primarily covers the management of this disorder, focusing on standard and innovative therapies, as well as on different approaches of administration.Expert opinion: The aim of treatment is to correct the dual defect of hemostasis caused by abnormal/reduced VWF and concomitant deficiency of factor VIII (FVIII). The therapy is indicated for management of bleeding and for short-term (i.e., surgical or invasive procedure) or long-term prophylaxis. The synthetic vasopressin analogue desmopressin (DDAVP) is the treatment of choice for about 80% of VWD patients, whereas replacement therapy with FVIII/VWF concentrates is indicated for those who are unresponsive to DDAVP.