DDAVP response and its determinants in bleeding disorders: a systematic review and meta-analysis
Blood, vol. 145, pp. 1814–1825
Abstract
ABSTRACT: Desmopressin (1-desamino-8-d-arginine vasopressin [DDAVP]) can be used to prevent or stop bleeding. However, large interindividual variability is observed in DDAVP response and determinants are largely unknown. In this systematic review and meta-analysis, we aimed to identify the response to DDAVP and the factors that determine DDAVP response in patients. We included studies with patients with any bleeding disorder receiving DDAVP. First and second screening round and risk of bias assessment were performed by independent reviewers. The main outcome was proportion of patients with complete (factor level >50 U/dL) or partial (30-50 U/dL) response to DDAVP. Determinants of response including disease type, age, sex, von Willebrand factor (VWF) and factor VIII (FVIII) mutations, and baseline factor levels were investigated. In total, 591 articles were found and 103 were included. Of these, 71 articles (1772 patients) were suitable for the study's definition of response. Meta-analysis showed a pooled response proportion of 0.71 (0.64; 0.78) and a significant difference in response between disease subtypes. For hemophilia A, baseline FVIII activity (FVIII:C) was a borderline significant determinant of response. In patients with von Willebrand disease (VWD) type 1, VWF antigen (VWF:Ag), VWF activity, and FVIII:C were significant determinants. A large variation in response was observed for specific mutations in VWF and FVIII. Response to DDAVP varied between disease subtypes and was largely determined by the baseline levels of FVIII:C for hemophilia A and VWF:Ag for VWD. Our findings highlight the significant differences in response and emphasize the need for a standardized response definition and further research into response mechanisms.
Authors 9
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Sebastiaan N. J. Laan corresponding Aachen 1Department of Internal Medicine Division of Thrombosis and Hemostasis
Leiden University · Leiden University Medical Center · Erasmus MC
Affiliation as printed
1Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden Universiteit Medical Centre, Leiden, The Netherlands
2Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
Leiden University Medical Center, Leiden, Netherlands
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Affiliation as printed
3Department of Molecular Hemostasis, Sanquin Research, Amsterdam, The Netherlands
Sanquin Research, Amsterdam, Netherlands
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Suzanne C. Cannegieter Aachen 1Department of Internal Medicine Division of Thrombosis and Hemostasis 4Department of Clinical Epidemiology
Leiden University · Leiden University Medical Center
Affiliation as printed
1Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden Universiteit Medical Centre, Leiden, The Netherlands
4Department of Clinical Epidemiology, Leiden University Medical Centre, Leiden, The Netherlands
Leiden University Medical Center, Leiden, Netherlands
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Sanquin · Amsterdam University Medical Centers
Affiliation as printed
3Department of Molecular Hemostasis, Sanquin Research, Amsterdam, The Netherlands
5Department of Pediatric Hematology, Amsterdam University Medical Centre, Amsterdam, The Netherlands
Sanquin Research, Netherlands
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Affiliation as printed
2Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
Erasmus University Medical Center, Rotterdam, Netherlands
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Saskia le Cessie Aachen 4Department of Clinical Epidemiology 6Department of Biomedical Data Sciences
Leiden University · Leiden University Medical Center
Affiliation as printed
4Department of Clinical Epidemiology, Leiden University Medical Centre, Leiden, The Netherlands
6Department of Biomedical Data Sciences, Leiden University Medical Centre, Leiden, The Netherlands
Leiden university medical centre, Leiden, Netherlands
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Affiliation as printed
2Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
Erasmus University Medical Center, Rotterdam, Netherlands
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Leiden University · Leiden University Medical Center
Affiliation as printed
1Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden Universiteit Medical Centre, Leiden, The Netherlands
Leiden University Medical Center, Leiden, Netherlands
-
Affiliation as printed
2Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
Erasmus University Medical Center, Rotterdam, Netherlands
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