去氨加压素治疗非重度甲型血友病：患者对使用及疗效的看法
Desmopressin in nonsevere hemophilia A: patient perspectives on use and efficacy.
作者信息
Romano Lorenzo G R, van Vulpen Lize F D, den Exter Paul L, Heubel-Moenen Floor C J I, Hooijmeijer Helene L, Coppens Michiel, Fijnvandraat Karin, Schols Saskia E M, Ypma Paula F, Smit Cees, Driessens Mariëtte H E, Rosendaal Frits R, van der Bom Johanna G, Gouw Samantha C, Kruip Marieke J H A
机构信息
Department of Hematology, Hemophilia Treatment Center, Erasmus MC, Erasmus University Medical Center, Rotterdam, The Netherlands.
Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
出版信息
Res Pract Thromb Haemost. 2023 Jun 17;7(5):100281. doi: 10.1016/j.rpth.2023.100281. eCollection 2023 Jul.
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.
背景
去氨加压素可提高非重度甲型血友病患者的血浆凝血因子 VIII 和血管性血友病因子水平。患者对去氨加压素的看法对于增加和优化其未充分利用的情况具有重要意义。然而，尚未有患者对去氨加压素看法的相关报道。
目的
评估非重度甲型血友病患者对去氨加压素使用的看法、使用障碍、副作用以及他们对去氨加压素疗效和副作用的了解情况。
方法
非重度甲型血友病患者纳入一项全国性、多中心横断面研究。成年患者及≥12 岁儿童自行填写问卷。<12 岁儿童由照料者填写问卷。
结果
共纳入 706 例非重度甲型血友病患者（544 例轻度，162 例中度，年龄范围 0 - 88 岁）。508 例患者中，234 例（50%）报告曾使用过去氨加压素。187 例患者中有 171 例（90%）认为去氨加压素至少有中度疗效。182 例患者中有 138 例（76%）选择鼻内给药。206 例成人中有 54 例（26%）、22 例儿童中有 7 例（32%）报告潮红是最常见的副作用。最常报告的优点和缺点分别是鼻内给药方便，可在院外使用，占 56%（126/227）；以及副作用，占 18%（41/227）。28%（63/225）的患者自我感觉对相关知识了解不足或不了解。
结论
总体而言，去氨加压素最常通过鼻内使用，且被认为有效，潮红是最常见的副作用。最常提到的优点是鼻内给药方便，缺点是有副作用。关于去氨加压素的更多信息和教育可以满足当前或未来接受去氨加压素治疗患者未满足的需求。
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