Narrative review evaluates the clinical profile of recombinant von Willebrand factor in von Willebrand disease, highlighting its potential for personalized bleeding disorder management.
INTRODUCTION: Von Willebrand disease (VWD) is the most common autosomally inherited bleeding disorder, characterized by a quantitative or qualitative defect of the multimeric adhesive glycoprotein von Willebrand factor (VWF). The mainstay of treatment of bleeding in VWD involves the use of desmopressin and plasma-derived factor VIII (FVIII)-VWF concentrates. In addition, a new recombinant VWF has been recently manufactured and licensed in the USA. AREAS COVERED: This narrative review, after a brief presentation of the current therapeutic strategies in VWD, will focus on recombinant VWF, analyzing its characteristics and the results of the completed phase I and III trials. Finally, the potential role of this recombinant drug in the therapeutic scenario of VWD is discussed. Expert commentary: Based on the evidence from literature analysis, we can conclude that recombinant VWF is a novel, interesting therapeutic option for VWD, which could help to further personalize the management of this complex inherited bleeding disorder.
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Franchini et al. (2016) studied this question.
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