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Thromb Haemost 2010; 104(05): 984-989
DOI: 10.1160/TH10-04-0220
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

Biological and clinical response to desmopressin (DDAVP) in a retrospective cohort study of children with low von Willebrand factor levels and bleeding history

Analía Sánchez-Luceros
1   Thrombosis and Haemostasis Departament, Haematological Research Institute, National Academy of Medicine, Buenos Aires, Argentina
2   National Research Council, CONICET, Ministry of Science, Technology and Productive Innovation, MINCyT, Buenos Aires, Argentina
,
Susana S. Meschengieser
1   Thrombosis and Haemostasis Departament, Haematological Research Institute, National Academy of Medicine, Buenos Aires, Argentina
,
Adriana I. Woods
2   National Research Council, CONICET, Ministry of Science, Technology and Productive Innovation, MINCyT, Buenos Aires, Argentina
,
Roberto Chuit
3   Epidemiologic Research Institute, National Academy of Medicine, Buenos Aires, Argentina
,
Karina Turdó
1   Thrombosis and Haemostasis Departament, Haematological Research Institute, National Academy of Medicine, Buenos Aires, Argentina
,
Alicia Blanco
1   Thrombosis and Haemostasis Departament, Haematological Research Institute, National Academy of Medicine, Buenos Aires, Argentina
,
María A. Lazzari
1   Thrombosis and Haemostasis Departament, Haematological Research Institute, National Academy of Medicine, Buenos Aires, Argentina
2   National Research Council, CONICET, Ministry of Science, Technology and Productive Innovation, MINCyT, Buenos Aires, Argentina
› Author Affiliations Financial support: This work was supported by grants from the CONICET, Agencia Nacional de Promoción Científica y Tecnológica (ANPCyT, MINCyT), Fundación Rene Barón and Academia Nacional de Medicina, Buenos Aires, Argentina.
Further Information

Publication History

Received: 10 April 2010

Accepted after major revision: 15 July 2010

Publication Date:
24 November 2017 (online)

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Summary

The diagnosis and management of von Willebrand disease (VWD) in paediatrics is challenging. Our aim was to review patient’s characteristics related to biological and clinical response to DDAVP in children with low von Willebrand factor (VWF) levels and bleeding history from a single institution. We included a retrospective cohort of 221 children (median age 11 years; 137 females): 27 type 1 (VWF levels within 15–30 IU dL-1) and 194 possible type 1 (VWF levels within 31–49 IU dL-1). The DDAVP infusion-test was performed in 214/221 children, 93.4% of whom showed good response. Patients with type 1 were at higher risk of DDAVP-test failure: 9/26 (34.6%) vs. 18/188 (9.6%) with possible type 1 (RR 3.44, 1.75–6.79; p= 0.002, Fisher’s exact test). In 68 children, the clinical response to DDAVP was evaluated 87 times: i) to stop bleeding: menorrhagia (13), mucocutaneous (12), haemarthrosis (1); and ii) to prevent surgical bleeding: adenotonsillectomy (17), major (15) and minor surgery (10); and dental procedures (19). No major adverse events or bleeding were observed. The treatment was effective with one single dose of DDAVP in almost all patients, without antifibrinolytic or local therapy, except in a girl with severe haemorrhage during menarche who required replacement therapy. In conclusion, patients with VWD type 1 were at higher risk of no response to DDAVP infusion-test. In this series, one dose of DDAVP proved effective and safe for children with VWD. Since this is a safe, effective and affordable therapy, we consider that a wider use should be promoted, especially in developing countries.

Keywords

Biological response - clinical response - children - desmopressin - von Willebrand disease
 

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