TY - JOUR
T1 - Desmopressin in nonsevere hemophilia A
T2 - patient perspectives on use and efficacy
AU - Romano, Lorenzo G. R.
AU - van Vulpen, Lize F. D.
AU - den Exter, Paul L.
AU - Heubel-Moenen, Floor C. J. I.
AU - Hooijmeijer, Helene L.
AU - Coppens, Michiel
AU - Fijnvandraat, Karin
AU - Schols, Saskia E. M.
AU - Ypma, Paula F.
AU - Smit, Cees
AU - Driessens, Mariette H. E.
AU - Md, Frits R. Rosendaal
AU - van der Bom, Johanna G.
AU - Gouw, Samantha C.
AU - Kruip, Marieke J. H. A.
PY - 2023/7/1
Y1 - 2023/7/1
N2 - 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 & GE;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.
AB - 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 & GE;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.
KW - advantages
KW - desmopressin
KW - hemophilia A
KW - patient perspective
KW - side effects
KW - survey
KW - QUALITY-OF-LIFE
KW - ENDOTHELIAL-CELLS
KW - FACTOR-VIII
KW - MILD
KW - EDUCATION
KW - DETERMINANTS
KW - KNOWLEDGE
KW - IMPACT
U2 - 10.1016/j.rpth.2023.100281
DO - 10.1016/j.rpth.2023.100281
M3 - Article
C2 - 37601028
SN - 2475-0379
VL - 7
JO - Research and practice in thrombosis and haemostasis
JF - Research and practice in thrombosis and haemostasis
IS - 5
M1 - 100281
ER -