Desmopressin in nonsevere hemophilia A: patient perspectives on use and efficacy

Lorenzo G R Romano; Lize F D van Vulpen; Paul L den Exter; Floor C J I Heubel-Moenen; Helene L Hooijmeijer; Michiel Coppens; Karin Fijnvandraat; Saskia E M Schols; Paula F Ypma; Cees Smit; Mariëtte H E Driessens; Frits R Rosendaal; Johanna G van der Bom; Samantha C Gouw; Marieke J H A Kruip

doi:10.1016/j.rpth.2023.100281

Desmopressin in nonsevere hemophilia A: patient perspectives on use and efficacy

Res Pract Thromb Haemost. 2023 Jun 17;7(5):100281. doi: 10.1016/j.rpth.2023.100281. eCollection 2023 Jul.

Authors

Lorenzo G R Romano¹, Lize F D van Vulpen², Paul L den Exter³, Floor C J I Heubel-Moenen⁴, Helene L Hooijmeijer⁵, Michiel Coppens^{6

7}, Karin Fijnvandraat^{8

9}, Saskia E M Schols¹⁰, Paula F Ypma¹¹, Cees Smit¹², Mariëtte H E Driessens¹², Frits R Rosendaal¹³, Johanna G van der Bom¹³, Samantha C Gouw^{8

13}, Marieke J H A Kruip¹

Affiliations

¹ Department of Hematology, Hemophilia Treatment Center, Erasmus MC, Erasmus University Medical Center, Rotterdam, The Netherlands.
² Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
³ Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, The Netherlands.
⁴ Department of Hematology, Maastricht University Medical Center+, Maastricht, The Netherlands.
⁵ Department of Pediatrics, University Medical Center Groningen, Groningen, The Netherlands.
⁶ Department of Vascular Medicine, Amsterdam University Medical Center, Amsterdam, The Netherlands.
⁷ Pulmonary Hypertension & Thrombosis, Amsterdam Cardiovascular Sciences, Amsterdam, The Netherlands.
⁸ Department of Pediatric Hematology, Amsterdam University Medical Center - Emma Children's Hospital, University of Amsterdam, Amsterdam, The Netherlands.
⁹ Department of Molecular Cellular Hemostasis, Sanquin Research and Landsteiner Laboratory, Amsterdam, The Netherlands.
¹⁰ Department of Hematology, Radboud University Medical Center and Hemophilia Treatment Center, Nijmegen-Eindhoven-Maastricht, Nijmegen, The Netherlands.
¹¹ Department of Hematology, Haga Hospital, The Hague, The Netherlands.
¹² Netherlands Hemophilia Patient Society (NVHP), Nijkerk, The Netherlands.
¹³ Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.

Abstract

Background: Desmopressin increases plasma factor VIII and von Willebrand factor levels in persons with nonsevere hemophilia A. Patients' perspectives on desmopressin are relevant to increase and optimize its suboptimal use. However, patients' views on desmopressin are not reported.

Objectives: To evaluate the perspectives of persons with nonsevere hemophilia A on desmopressin use, barriers for its use, side effects, and their knowledge about desmopressin's efficacy and side effects.

Methods: Persons with nonsevere hemophilia A were included in a cross-sectional, national, multicenter study. Questionnaires were filled out by adult patients and children aged ≥12 years themselves. Caretakers filled out questionnaires for children aged <12 years.

Results: In total, 706 persons with nonsevere hemophilia A were included (544 mild, 162 moderate, [age range, 0-88 years]). Of 508 patients, 234 (50%) patients reported previous desmopressin use. Desmopressin was considered as at least moderately effective in 171 of 187 (90%) patients. Intranasal administration was the modality of choice for 138 of 182 (76%) patients. Flushing was the most reported side effect in 54 of 206 (26%) adults and 7 of 22 (32%) children. The most frequently reported advantage and disadvantage were the convenience of intranasal, out-of-hospital administration by 56% (126/227) and side effects in 18% (41/227), respectively. Patients' self-perceived knowledge was unsatisfactory or unknown in 28% (63/225).

Conclusion: Overall, desmopressin was most often used intranasally and considered effective, with flushing as the most common side effect. The most mentioned advantage was the convenience of intranasal administration and disadvantage was side effects. More information and education on desmopressin could answer unmet needs in patients with current or future desmopressin treatment.

Keywords: advantages; desmopressin; hemophilia A; patient perspective; side effects; survey.