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Blood, 15 March 2004, Vol. 103, No. 6, pp. 2032-2038. Prepublished online as a Blood First Edition Paper on November 20, 2003; DOI 10.1182/blood-2003-06-2072. This Article Full Text (PDF) All Versions of this Article: 2003-06-2072v1 103/6/2032    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Federici, A. B Articles by Mannucci, P. M Search for Related Content PubMed PubMed Citation Articles by Federici, A. B Articles by Mannucci, P. M Related Collections Related Article in Blood Online Social Bookmarking                   What's this? Submitted June 25, 2003 Accepted November 10, 2003 Biological response to desmopressin in patients with severe type 1 and type 2 von Willebrand disease: results of a multicenter European study Augusto B Federici, Claudine Mazurier, Erik Berntorp, Christine A Lee, Inge Scharrer, Jenny Goudemand, Stefan Lethagen, Ioana Nitu, Gerard Ludwig, Lysiane Hilbert, and Pier M Mannucci* IRCCS Maggiore Hospital and University of Milan, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy Laboratoire Francais du Fractionnement et des Biotechnologies, Lille, France Malmo Univeristy Hospital, Department for Coagulation Disorders, Malmo, Sweden Royal Free Hospital, Hemophilia Centre and Hemostasis Unit, London, United Kingdom Medical Department 1, J. W. Goethe University Hospital Hemophilia and Thrombosis Center, Frankfurt, Germany Hopital Claude Hirez, Laboratoire d'Hematologie, Lille, France * Corresponding author; email: pmmannucci{at}libero.it. This study prospectively evaluated the rate of biological response to desmopressin (DDAVP) in 66 patients with type 1 or 2 von Willebrand disease (VWD) who on the basis of available records had a clinically significant bleeding history and at least one of the following laboratory abnormalities: bleeding time (BT) >15 min, ristocetin cofactor activity (VWF:RCo) <10 IU/dL, factor VIII coagulant activity (FVIII:C) <20 IU/dL ("severe" VWD). Prior to the study, responsive patients were defined as those who, two hours after infusion of 0.3 µg/kg DDAVP, increased their baseline VWF:RCo and FVIII:C by at least three fold, and achieved levels of both measurements of at least 30 IU/dl, and a BT of 12 min. The rate of biological response varied according to VWD types and was higher in type 1 (7/26,27%) than in type 2 (7/40,18%) [type 2A (1/15,7%), type 2M (3/21,14%), type 2N (3/4,75%)]. Mutations in the VWF gene were previously known or newly identified in the majority of patients with types 2A (n=15/15), 2M (n=15/21) and 2N (n=4/4), but in none of those with type 1 VWD. Genotype provided more information than phenotype to predict the individual response to DDAVP only in patients with 2A and 2N VWD. This prospective study showed that the rate of biological response to DDAVP is relatively low not only in type 2 but also in type 1 VWD when uniform and stringent criteria for patient selection and responsiveness are applied. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Article in Blood Online: Responsiveness of patients with VWD to DDAVP Robert R. Montgomery Blood 2004 103: 1975-1976. [Full Text] [PDF] This article has been cited by other articles: F. Rodeghiero, G. Castaman, and A. Tosetto How I treat von Willebrand disease Blood, August 6, 2009; 114(6): 1158 - 1165. [Abstract] [Full Text] [PDF] A. B Federici and M. T. 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