Biological therapies for von Willebrand disease

von Willebrand factor (VWF)-related disorders are generally distinguished as either acquired (von Willebrand Syndrome; VWS) or congenital (von Willebrand Disease; VWD). VWD is the most common inherited bleeding disorder and is due to deficiencies and/or defects in VWF. VWS arises from a large variety of causes.

The current report briefly overviews the diagnosis of VWD and VWS, but primarily covers the management of these disorders, as reflected by differential processes applied within different centers worldwide, also focusing on emerging trends in biological therapies. Most developed countries currently use standard therapy to manage bleeding, employing desmopressin wherever possible, factor concentrate in other situations and additional (e.g., antifibrinolytic) therapy when required. With regards to factor concentrates, there are differences in content between those available in relation to levels and composition of VWF and factor VIII, and only selective concentrates are available in different localities.

All these aspects reflect important but sometimes overlooked issues when using replacement therapy, or attempting to follow expert guidelines. Recombinant VWF has been developed and is undergoing clinical trials, and this promising therapy may change the VWD management landscape in the near future.