药物动力学导向与血友病的标准预防:系统性审查和元分析
Daniel Kraemmer1, Oliver Königsbrügge1, Florian Moik2
1Clinical Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
Journal of thrombosis and haemostasis : JTH
|September 22, 2023
概括
目前在血友病中PK导向预防的证据很弱. 一项系统性审查发现,相对于标准预防,临床益处有限,主要是由于非随机研究中的偏差.
科学领域:
- 血液学 血液学 血液学
- 药理动力学 药理动力学
- 基于证据的医学 基于证据的医学
背景情况:
- 人口药理动力学 (PK) 建模越来越多.
- 目前的血友病指南支持PK导向的预防.
- 进行了系统审查,以评估现有证据.
研究的目的:
- 评估与非PK引导的预防相比,PK引导的预防的有效性.
- 总结关于在血友病中使用PK导向预防的当前文献.
主要方法:
- 对多个数据库 (MEDLINE,Embase,CENTRAL,ClinicalTrials.gov,欧盟临床试验注册) 的系统审查.
- 包括所有研究的PK导向预防队列.
- 使用随机效应模型进行的随机临床试验 (RCT) 的研究和定量综合的定性总结.
主要成果:
- 25项研究符合纳入标准,2项RCT和17项非随机研究具有比较组.
- 非随机化研究具有相当大的偏差风险.
- 对2个RCT的元分析对出血结果和因子消耗没有确定性.
结论:
- 证据表明,PK导向预防对标准预防的临床益处较弱.
- 这些发现主要来自非随机研究,但存在重大局限性.
- 需要进一步的高质量研究来证实有效性.
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