扩大以患者为中心的心血管临床试验终点的使用和解释
Shelby D Reed1, Pishoy Gouda1,2
1Duke Clinical Research Institute, Duke University, Durham, NC, USA.
Journal of clinical and translational science
|September 2, 2025
概括
新的临床试验终点通过测量对患者重要的结果来提高患者中心性. 有各种方法可以结合这些结果,但需要更多的研究来比较它们的含义.
科学领域:
- 临床研究方法
- 患者报告的结果
- 卫生经济学与成果研究
背景情况:
- 提高临床研究的患者中心性对于将试验结果与患者的价值观和经验保持一致至关重要.
- 越来越多地使用新的临床试验终点,例如活出和出院日 (DAOH) 和调整后的生命质量结果.
- 综合结果,包括患者重要的事件和偏好,提供更丰富的治疗益处评估.
研究的目的:
- 探索以患者为中心的临床试验终点的开发和利用.
- 讨论构建和分析综合患者中心结果的细微差别和方法.
- 强调需要对以患者为中心的结果分析的不同方法进行比较研究.
主要方法:
- 重点关注患者报告的结果和实践经验的新临床试验终点方法的审查.
- 综合结果构造的讨论,包括事件选择,结果定义和权重的提取.
- 分析方法的概述,用于结合加权结果,例如胜率,加权胜率,DOOR和MCDA.
主要成果:
- 以患者为中心的终点捕捉了与患者健康状况和日常生活直接相关的结果.
- 综合结果方法允许整合多个,排序或加权事件.
- 为了分析以患者为中心的综合结果,存在各种统计方法 (win ratio,DOOR,MCDA),每种方法都有特定的优势和挑战.
结论:
- 改善临床研究中以患者为中心的结果是患者,临床医生和监管机构的关键目标.
- 在综合结果中选择和加权事件需要仔细考虑患者的偏好和临床相关性.
- 需要进一步进行比较研究,以了解各种以患者为中心的结果分析方法的含义和最佳应用.
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