Prophylaxis in von Willebrand disease.
Von Willebrand disease (VWD), the most common hereditary bleeding disorder, is divided into three types depending on the quantitative (type 1 and 3) or qualitative (type 2) abnormality of von Willebrand factor (VWF). About 70-80% of VWD patients can be treated with the synthetic product desmopressin, while the others necessitate factor VIII/VWF concentrates. In addition to the treatment of bleeding episodes, therapeutic regimens include short- or long-term prophylaxis. While the literature data on short-term prophylaxis in VWD are consistent and clearly show the safety and efficacy of such a therapeutic approach, little evidence is available regarding long-term prophylaxis, and although the preliminary results are encouraging, they need to be validated by large prospective studies.
Franchini
Massimo
M
Servizio di Immunoematologia e Trasfusione, Centro Emofilia, Azienda Ospedaliera di Verona, Verona, Italy.
Targher
Giovanni
G
Lippi
Giuseppe
G
D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use*; D005169:Factor VIII / Q000378:metabolism / Q000627:therapeutic use*; D006490:Hemostatics / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011446:Prospective Studies; D013997:Time Factors; D014842:von Willebrand Diseases / Q000188:drug therapy / Q000378:metabolism / Q000517:prevention & control*; D014841:von Willebrand Factor / Q000378:metabolism / Q000627:therapeutic use*
D006490:Hemostatics; D014841:von Willebrand Factor; D005169:Factor VIII; D003894:Deamino Arginine Vasopressin