Prophylaxis of bleeding episodes in patients with von Willebrand's disease.
Patients with severe forms of von Willebrand's disease (VWD) may have frequent haemarthroses, especially when factor VIII (FVIII) levels are below 10 U/dL, so that some of them develop target joints like patients with severe haemophilia A. Some patients have recurrent gastrointestinal bleeding, often without lesions in the gastrointestinal tract, and need treatment every day or every other day. Finally, there are children who have epistaxis frequently and severely enough to cause anaemia. In these frequent and severe bleeders, the optimal therapy may be secondary long-term prophylaxis with von Willebrand factor (VWF)/FVIII concentrates rather than on-demand treatment on the occasion of bleeding episodes. The largest experience on such prophylaxis in VWD has been in Sweden in 35 patients with severe forms of VWD. Long-term prophylaxis was also implemented in a cohort of Italian patients with VWD: prophylaxis was used in seven patients with types 3 (n = 1 ), 2A (n = 4), 2M (n = 1) and type 1 (n = 1) VWD because of recurrent gastrointestinal bleeds and in four patients with type 3 VWD because of joint bleeds. Prophylaxis prevented bleeding completely in eight patients and largely reduced hospitalisation for blood transfusions in the remaining three. The cost-effectiveness of these prophylaxis regimens versus on-demand therapy will now be investigated in one large international study
Federici
Augusto B
AB
Centro Emofilia e Trombosi Angelo Bianchi Bonomi, Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena, Università degli Studi di Milano, Milano, Italia. augusto.federici@unimi.it
D002986:Clinical Trials as Topic / Q000706:statistics & numerical data; D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004338:Drug Combinations; D004844:Epistaxis / Q000209:etiology / Q000517:prevention & control; D005169:Factor VIII / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D005340:Fibrinogen / Q000627:therapeutic use; D006471:Gastrointestinal Hemorrhage / Q000209:etiology / Q000517:prevention & control; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D015337:Multicenter Studies as Topic / Q000706:statistics & numerical data; D011446:Prospective Studies; D012189:Retrospective Studies; D055502:Secondary Prevention; D014842:von Willebrand Diseases / Q000150:complications / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000008:administration & dosage / Q000627:therapeutic use*
D004338:Drug Combinations; C026912:cryoprecipitate coagulum; C509423:factor VIII, von Willebrand factor drug combination; D014841:von Willebrand Factor; D005169:Factor VIII; D005340:Fibrinogen; D003894:Deamino Arginine Vasopressin