Zwagemaker Anne-Fleur, Kloosterman Fabienne R, Coppens Michiel, Gouw Samantha C, Boyce Sara, Bagot Catherine N, Beckers Erik A M, Brons Paul, Castaman Giancarlo, Eikenboom Jeroen, Jackson Shannon, Kruip Marieke J H A, Leebeek Frank W G, Meijer Karina, Nieuwenhuizen Laurens, Pabinger Ingrid, Fijnvandraat Karin
Amsterdam UMC University of Amsterdam, Emma Children's Hospital, Pediatric Hematology Amsterdam The Netherlands.
Department of Vascular Medicine Amsterdam University Medical Centers Amsterdam The Netherlands.
Res Pract Thromb Haemost. 2022 Aug 31;6(6):e12777. doi: 10.1002/rth2.12777. eCollection 2022 Aug.
Desmopressin is an important treatment option in nonsevere hemophilia A because it has several benefits compared with factor (F) concentrates, including no inhibitor risk and much lower costs. Despite these advantages, data are limited on the real-world use of desmopressin in the treatment of bleeds.
To describe the clinical use of desmopressin in relation to other therapeutic modalities in the treatment of bleeding episodes in patients with nonsevere hemophilia A.
Patients with nonsevere hemophilia A aged 12-55 years were included from the DYNAMO cohort study. Data on the desmopressin test response and treated bleeding events in the period January 2009 to July 2020 were retrospectively collected from medical files. An adequate desmopressin test response was defined based on a peak FVIII level of ≥30 IU/dl.
A total of 248 patients with a median age of 38 years (interquartile range 25-49) were included. An adequate desmopressin test response was documented in 25% and 73% of patients with moderate and mild hemophilia, respectively. In adequate responders, 51% of bleeds were exclusively treated with FVIII concentrates, 24% exclusively with desmopressin, 21% with a combination of both and 4% with other treatments. In 54% of bleeds treated with a single dose of factor concentrates, the expected FVIII level after desmopressin exceeded the level targeted.
Most bleeds in patients with an adequate response to desmopressin are treated with factor concentrates. These findings may indicate a suboptimal use of desmopressin and that barriers to the use of desmopressin should be explored.
去氨加压素是治疗非重度甲型血友病的重要选择，因为与凝血因子（F）浓缩物相比，它有诸多益处，包括无抑制剂风险且成本低得多。尽管有这些优势，但关于去氨加压素在出血治疗中的实际应用数据有限。
描述去氨加压素在治疗非重度甲型血友病患者出血发作时与其他治疗方式相关的临床应用。
从DYNAMO队列研究中纳入年龄在12 - 55岁的非重度甲型血友病患者。回顾性收集2009年1月至2020年7月期间去氨加压素试验反应及治疗出血事件的数据，来自医疗档案。基于FVIII峰值水平≥30 IU/dl定义去氨加压素试验反应充分。
共纳入248例患者，中位年龄38岁（四分位间距25 - 49）。中度和轻度血友病患者中分别有25%和73%记录到去氨加压素试验反应充分。在反应充分的患者中，51%的出血仅用FVIII浓缩物治疗，24%仅用去氨加压素治疗，21%两者联合使用，4%用其他治疗。在单次剂量凝血因子浓缩物治疗的出血中，54%的患者去氨加压素后的预期FVIII水平超过目标水平。
对去氨加压素反应充分的患者，大多数出血用凝血因子浓缩物治疗。这些发现可能表明去氨加压素使用未达最佳，应探索去氨加压素使用的障碍。
