Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Medical Centre, Alberta Health Services, Calgary, Alberta, Canada.
University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada.
Haemophilia. 2018 Sep;24(5):720-725. doi: 10.1111/hae.13586. Epub 2018 Jul 13.
Desmopressin is an effective haemostatic agent for patients with non-severe haemophilia A; however, response may differ between patients of similar severity. Responsiveness is classified based on various cut-off values for plasma levels of FVIII post-desmopressin administration. Patients may be classified differently depending on the values chosen.
To classify desmopressin response in non-severe haemophilia A patients with respect to current test-response definitions. Also, to characterize relationships between test response and clinical outcome of desmopressin use.
Current desmopressin test-response definitions were obtained from the literature. We adopted peak FVIII level (at 1 hour post-administration) ≥50 IU/dL and <20 IU/dL as complete and no response, respectively, thereby satisfying most reported definitions. Test-responses and clinical outcomes of use between 2007 and 2017 for adult mild/moderate haemophilia A patients were reviewed and correlated.
All patients classified as complete responders (n = 31; peak FVIII ≥50 IU/dL) and the majority of partial responders (n = 11; peak FVIII ≥20 to <50 IU/dL) had good clinical outcomes after desmopressin use for a variety of bleeding episodes and procedures. Two non-responders (peak FVIII <20 IU/dL) given desmopressin for minor bleeding/procedures also had good clinical outcomes. One patient with a partial test-response (peak FVIII 23 IU/dL) required additional factor concentrate to achieve haemostasis.
Based on our review, we suggest that the determination of desmopressin responsiveness should consider both the change in plasma FVIII levels as well as clinical outcomes associated with prior therapeutic use.
去氨加压素是治疗非重度甲型血友病患者的有效止血剂；然而，不同严重程度的患者之间的反应可能存在差异。反应性基于去氨加压素给药后 FVIII 血浆水平的各种截止值进行分类。根据所选值，患者可能会被分类为不同类型。
根据当前的测试反应定义，对非重度甲型血友病患者的去氨加压素反应进行分类。此外，还要描述测试反应与去氨加压素使用临床结果之间的关系。
从文献中获得当前的去氨加压素测试反应定义。我们采用峰值 FVIII 水平（给药后 1 小时）≥50IU/dL 和 <20IU/dL 分别作为完全和无反应，从而满足大多数报告的定义。回顾并比较了 2007 年至 2017 年期间成年轻度/中度甲型血友病患者的测试反应和使用临床结果。
所有被归类为完全反应者（n=31；峰值 FVIII ≥50IU/dL）和大多数部分反应者（n=11；峰值 FVIII ≥20 至 <50IU/dL）在接受去氨加压素治疗各种出血事件和手术时均取得了良好的临床结果。两名接受去氨加压素治疗轻微出血/手术的非反应者（峰值 FVIII <20IU/dL）也取得了良好的临床结果。一名部分测试反应者（峰值 FVIII 23IU/dL）需要额外的因子浓缩物来实现止血。
根据我们的审查，我们建议去氨加压素反应性的确定应同时考虑血浆 FVIII 水平的变化以及与先前治疗性使用相关的临床结果。
