Desmopressin in moderate hemophilia A patients: a treatment worth considering
Janneke I. Loomans,
Marieke J.H.A. Kruip,
Manuel Carcao,
Shannon Jackson,
Alice S. van Velzen,
Marjolein Peters,
Elena Santagostino,
Helen Platokouki,
Erik Beckers,
Jan Voorberg,
Johanna G. van der Bom,
Karin Fijnvandraat,
for the RISE consortium
Affiliations
Janneke I. Loomans
Department of Pediatric Hematology, Immunology and Infectious diseases, Emma Children’s Hospital, Amsterdam, the Netherlands
Marieke J.H.A. Kruip
Department of Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands
Manuel Carcao
Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada
Shannon Jackson
Division of Hematology, Department of Medicine, St. Paul’s Hospital and University of British Columbia, Vancouver, BC, Canada
Alice S. van Velzen
Department of Pediatric Hematology, Immunology and Infectious diseases, Emma Children’s Hospital, Amsterdam, the Netherlands
Marjolein Peters
Department of Pediatric Hematology, Immunology and Infectious diseases, Emma Children’s Hospital, Amsterdam, the Netherlands
Elena Santagostino
A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy
Helen Platokouki
Aghia Sofia Children’s Hospital, Athens, Greece
Erik Beckers
Maastricht University Medical Centre, the Netherlands
Jan Voorberg
Department of Plasma Proteins, Sanquin Research, Amsterdam, the Netherlands
Johanna G. van der Bom
Leiden University Hospital, the Netherlands;Sanquin Research, Leiden, the Netherlands
Karin Fijnvandraat
Department of Pediatric Hematology, Immunology and Infectious diseases, Emma Children’s Hospital, Amsterdam, the Netherlands;Department of Plasma Proteins, Sanquin Research, Amsterdam, the Netherlands
Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response, therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though factor VIII levels may rise substantially in some of them. We aim to describe the response to desmopressin in moderate hemophilia A patients and to identify predictors. We selected data on 169 patients with moderate hemophilia from the multicenter Response to DDAVP In non-severe hemophilia A patients: in Search for dEterminants (RISE) cohort study. Adequate response to desmopressin was defined as a peak factor VIII level ≥ 30, and excellent response as ≥ 50 international units/deciliter after desmopressin administration. We used univariate and multiple linear regression techniques to analyze predictors of the peak factor VIII level. Response was considered adequate in 68 patients (40%), of whom 25 showed excellent response (15%). Intravenous administration, age, pre-desmopressin factor VIII activity and von Willebrand factor antigen, peak von Willebrand factor activity and desmopressin-induced rise in von Willebrand factor antigen were significant predictors of peak factor VIII level and explained 65% of the inter-individual variation. In 40% of moderate hemophilia A patients, desmopressin response was adequate, thus it is important not to with-hold this group of patients from desmopressin responsiveness. Among the six predictors that we identified for desmopressin-induced factor VIII rise, factor VIII activity and desmopressin-induced rise in von Willebrand factor antigen had the strongest effect.
