title: Prophylaxis in von Willebrand Disease: Coming of Age?
abstract: Although in most cases von Willebrand disease (VWD) is a mild disorder, a subgroup of patients experience frequent bleeding. In contrast to severe hemophilia in which prophylaxis is the accepted standard of care, this is less frequently used in VWD. Most type 1 VWD patients can be adequately managed with episodic desmopressin and tranexamic acid. In patients with more severe disease, especially those with type 3 VWD, joint bleeds, epistaxis, menorrhagia, and gastrointestinal bleeding are problematic and usually require treatment with von Willebrand factor/factor VIII (VWF/FVIII) concentrate. While in the past these patients were managed with on-demand VWF/FVIII concentrate, several recent reports have demonstrated the value of prophylactic treatment. Despite some uncertainties about the economic impact of treatment of severe VWD, prophylaxis with VWF concentrate should now be considered as the standard of care for the more severe end of the spectrum of affected individuals. The recent introduction of recombinant VWF concentrate is likely to improve the acceptability of prophylaxis in VWD.
authors.affiliation: Centro di Riferimento Regionale per le Coagulopatie Congenite del bambino e dell'adulto, U.O. Ematologia con TMO, Policlinico Paolo Giaccone, Palermo, Italia.
authors.forename: Giorgia
authors.initials: G
authors.lastname: Saccullo
authors.affiliation: Sheffield Haemophilia and Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, United Kingdom.
authors.forename: Mike
authors.initials: M
authors.lastname: Makris
mesh_terms: D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use*; D005169:Factor VIII / Q000627:therapeutic use*; D006801:Humans; D014148:Tranexamic Acid / Q000627:therapeutic use*; D056725:von Willebrand Disease, Type 1 / Q000517:prevention & control*; D056729:von Willebrand Disease, Type 3 / Q000517:prevention & control*; D014841:von Willebrand Factor / Q000627:therapeutic use*
chemical_list: D014841:von Willebrand Factor; D014148:Tranexamic Acid; C078147:F8 protein, human; D005169:Factor VIII; D003894:Deamino Arginine Vasopressin