|
|
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.
 Previous Article | Table of Contents | Next Article 
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Biologic 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,
Stephan Lethagen,
Ioana Nitu,
Gerard Ludwig,
Lysiane Hilbert, and
Pier M. Mannucci
From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Department of Internal Medicine and Dermatology, IRCCS Maggiore Hospital, and University of Milan, Italy; Laboratoire Français du Fractionnement et des Biotechnologies, Lille, France; Department for Coagulation Disorders, Malmö University Hospital, Sweden; Hemophilia Centre and Hemostasis Unit, Royal Free Hospital, London, United Kingdom; J.W. Goethe University Hospital Hemophilia and Thrombosis Center, Medical Department 1, Frankfurt/Mainz, Germany; and Laboratoire d'Hématologie, Hôpital Claude Huriez, Lille, France.
This study prospectively evaluated the rate of biologic response to desmopressin (DDAVP) in 66 patients with type 1 or 2 von Willebrand disease (VWD), each of whom had, on the basis of available records, a clinically significant bleeding history and at least one of the following laboratory abnormalities: bleeding time (BT) longer than 15 minutes, ristocetin cofactor activity (VWF:RCo) less than 10 IU/dL, factor VIII coagulant activity (FVIII:C) less than 20 IU/dL (severe VWD). Before the study, responsive patients were defined as those who, 2 hours after infusion of 0.3 µg/kg DDAVP, had increased baseline values of VWF:RCo and FVIII:C by at least 3-fold and achieved levels of at least 30 IU/dL for both and a BT of 12 minutes or less. The rate of biologic response varied according to VWD types and was higher in type 1 (7 of 26, 27%) than in type 2 (7 of 40, 18%) (type 2A [1 of 15, 7%], type 2M [3 of 21, 14%], type 2N [3 of 4, 75%]). Mutations in the VWF gene were previously known or newly identified in most patients with types 2A (n = 15 of 15), 2M (n = 15 of 21), and 2N (n = 4 of 4), but in none of those with type 1 VWD. Genotype provided more information than phenotype in predicting individual responses to DDAVP only in patients with 2A and 2N VWD. This prospective study showed that the rate of biologic 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:
|
|
|
|
 
S. L. Haberichter, G. Castaman, U. Budde, I. Peake, A. Goodeve, F. Rodeghiero, A. B. Federici, J. Batlle, D. Meyer, C. Mazurier, et al.
Identification of type 1 von Willebrand disease patients with reduced von Willebrand factor survival by assay of the VWF propeptide in the European study: Molecular and Clinical Markers for the Diagnosis and Management of Type 1 VWD (MCMDM-1VWD)
Blood,
May 15, 2008;
111(10):
4979 - 4985.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
G. Castaman, S. Lethagen, A. B. Federici, A. Tosetto, A. Goodeve, U. Budde, J. Batlle, D. Meyer, C. Mazurier, E. Fressinaud, et al.
Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD
Blood,
April 1, 2008;
111(7):
3531 - 3539.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. Giannini, A. M. Mezzasoma, M. Leone, and P. Gresele
Laboratory diagnosis and monitoring of desmopressin treatment of von Willebrand's disease by flow cytometry
Haematologica,
December 1, 2007;
92(12):
1647 - 1654.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. B. Federici, G. Castaman, M. Franchini, M. Morfini, E. Zanon, A. Coppola, A. Tagliaferri, E. Boeri, M. G. Mazzucconi, G. Rossetti, et al.
Clinical use of Haemate(R) P in inherited von Willebrand's disease: a cohort study on 100 Italian patients
Haematologica,
July 1, 2007;
92(7):
944 - 951.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
B. Ozgonenel, M. Rajpurkar, and J. M Lusher
How do you treat bleeding disorders with desmopressin?
Postgrad. Med. J.,
March 1, 2007;
83(977):
159 - 163.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. J. Michiels, H. H. D. M. van Vliet, Z. Berneman, A. Gadisseur, M. van der Planken, W. Schroyens, A. van der Velden, and U. Budde
Intravenous DDAVP and Factor VIII-von Willebrand Factor Concentrate for the Treatment and Prophylaxis of Bleedings in Patients With von Willebrand Disease Type 1, 2 and 3
Clinical and Applied Thrombosis/Hemostasis,
January 1, 2007;
13(1):
14 - 34.
[Abstract]
[PDF]
|
|
|
|
|
|
|
A. B. Federici
VWF propeptide: a useful marker in VWD
Blood,
November 15, 2006;
108(10):
3229 - 3230.
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. J. Michiels, Z. Berneman, A. Gadisseur, M. van der Planken, W. Schroyens, A. van de Velde, and H. van Vliet
Classification and Characterization of Hereditary Types 2A, 2B, 2C, 2D, 2E, 2M, 2N, and 2U (Unclassifiable) von Willebrand Disease.
Clinical and Applied Thrombosis/Hemostasis,
October 1, 2006;
12(4):
397 - 420.
[Abstract]
[PDF]
|
|
|
|
|
|
|
P. M. Mannucci
Treatment of von Willebrand's Disease
N. Engl. J. Med.,
August 12, 2004;
351(7):
683 - 694.
[Full Text]
[PDF]
|
|
|
| |
