The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease.
Analyses of the bleeding tendency by means of the bleeding score (BS) have been proposed until now to confirm diagnosis but not to predict clinical outcomes in patients with inherited von Willebrand disease (VWD). We prospectively followed up, for 1 year, 796 Italian patients with different types of VWD to determine whether the previous BS of European VWD1 is useful to predict the occurrence of spontaneous bleeds severe enough to require replacement therapy with desmopressin (DDAVP) and/or von Willebrand factor (VWF)/factor VIII concentrates. Among the 796 patients included, 75 (9.4%) needed treatment of 232 spontaneous bleeding events. BS >10 and VWF:ristocetin cofactor activity <10 U/dL were associated with the risk of bleeding, but only a BS >10 remained highly associated in a multivariable Cox proportional hazard model (adjusted hazard ratio: 7.27 [95% confidence interval, 3.83-13.83]). Although the bleeding event-free survival was different in VWD types, only a BS >10 could predict for each type which patient had bleeding events severe enough to require treatment with DDAVP and/or concentrates. Therefore, BS can be considered a simple predictor of clinical outcomes of VWD and may identify patients needing intensive therapeutic regimens.
Angelo Bianchi Bonomi Hemophilia Thrombosis Center, Istituto Ricerca e Cura di Carattere Scientifico (IRCCS) Ca' Granda, Foundation Maggiore Policlinico Hospital, Milan, Italy; Hematology and Transfusion Medicine, L. Sacco University Hospital and Department of Clinical Sciences & Community Health, University of Milan, Milan, Italy;
Augusto B
AB
Federici
Angelo Bianchi Bonomi Hemophilia Thrombosis Center, Istituto Ricerca e Cura di Carattere Scientifico (IRCCS) Ca' Granda, Foundation Maggiore Policlinico Hospital, Milan, Italy;
Paolo
P
Bucciarelli
Division of Hematology, San Bortolo Hospital, Vicenza, Italy;
Giancarlo
G
Castaman
Hemophilia Center, Department of Hematology, University of Rome, Rome, Italy;
Maria G
MG
Mazzucconi
Hemophilia Center, Careggi Hospital, Florence, Italy;
Massimo
M
Morfini
Hemophilia Center, Pellegrini Hospital, Naples, Italy;
Angiola
A
Rocino
Hemophilia Thrombosis Center, Ospedale Policlinico, Bari, Italy; and.
Mario
M
Schiavoni
Angelo Bianchi Bonomi Hemophilia Thrombosis Center, Istituto Ricerca e Cura di Carattere Scientifico (IRCCS) Ca' Granda, Foundation Maggiore Policlinico Hospital, Milan, Italy;
Flora
F
Peyvandi
Division of Hematology, San Bortolo Hospital, Vicenza, Italy;
Francesco
F
Rodeghiero
Scientific Direction, IRCCS Cà Granda Foundation Maggiore Policlinico Hospital, Milan, Italy.
Pier Mannuccio
PM
Mannucci
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D003894:Deamino Arginine Vasopressin / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005169:Factor VIII; D005260:Female; D005500:Follow-Up Studies; D006470:Hemorrhage / Q000188:drug therapy / Q000453:epidemiology / Q000401:mortality / Q000473:pathology; D006490:Hemostatics / Q000008:administration & dosage*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D012307:Risk Factors; D012720:Severity of Illness Index; D015996:Survival Rate; D014842:von Willebrand Diseases / Q000097:blood / Q000188:drug therapy / Q000401:mortality / Q000473:pathology; D014841:von Willebrand Factor / Q000008:administration & dosage*
D006490:Hemostatics; D014841:von Willebrand Factor; C078147:F8 protein, human; D005169:Factor VIII; D003894:Deamino Arginine Vasopressin