title: Von Willebrand factor (Vonvendi®): the first recombinant product licensed for the treatment of von Willebrand disease.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
authors.affiliation: a Department of Transfusion Medicine and Hematology , Carlo Poma Hospital , Mantova , Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Franchini
authors.affiliation: b Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico and University of Milan , Milan , Italy.
authors.forename: Pier Mannuccio
authors.initials: PM
authors.lastname: Mannucci
mesh_terms: D002986:Clinical Trials as Topic; D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use*; D016896:Treatment Outcome; D014842:von Willebrand Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology; D014841:von Willebrand Factor / Q000737:chemistry / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*
chemical_list: D011994:Recombinant Proteins; D014841:von Willebrand Factor; D005169:Factor VIII; D003894:Deamino Arginine Vasopressin
keywords: bleeding; recombinant von Willebrand factor; therapy; von Willebrand disease