Evidence-based recommendations on the treatment of von Willebrand disease in Italy.
BACKGROUND
von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII).


METHODS
This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease.


CONCLUSIONS
Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
Mannucci
Pier Mannuccio
PM
Centro Emofilia e Trombosi Angelo Bianchi Bonomi, Dipartimento di Medicina e Specialità Mediche, Università di Milano e IRCCS Fondazione Ospedale Maggiore, Italy. piermannuccio.mannucci@unimi.it
Franchini
Massimo
M
Castaman
Giancarlo
G
Federici
Augusto B
AB
D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D019317:Evidence-Based Medicine; D005169:Factor VIII / Q000627:therapeutic use*; D005260:Female; D006490:Hemostatics / Q000627:therapeutic use*; D006801:Humans; D007558:Italy; D008297:Male; D016896:Treatment Outcome; D014842:von Willebrand Diseases / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000627:therapeutic use*
D006490:Hemostatics; D014841:von Willebrand Factor; D005169:Factor VIII; D003894:Deamino Arginine Vasopressin