Historical, current and future treatments for von Willebrand disease

Von Willebrand disease (VWD) is a heterogeneous group of defects characterized by a spectrum of bleeding symptoms ranging from mild to severe, which remain difficult to identify and assess quantitatively. Despite significant advances in our understanding of the pathophysiology of the disease, diagnosis and management remain challenging. This review examines the therapeutic landscape for VWD, discussing historical treatments, recent advancements and prospects. Decades of clinical evidence supporting the efficacy of replacement therapy will be critically presented, and preclinical data for emerging options will be examined. For many years, the standard of care for VWD has involved replacement therapy with blood-derived products and desmopressin. The introduction of recombinant von Willebrand factor represents a more recent development compared to other recombinant factors, and its use in certain populations of patients is still under investigation. Despite being relatively new, innovative therapeutic options are being explored and developed to address patients' unmet needs. Some of these therapies are currently undergoing or nearing clinical evaluation, while others remain in the preclinical phase of development. After years of neglected attention, innovation in the treatment of VWD is now rapidly expanding.