在存在半竞争风险的情况下,直接和间接的治疗效应
Yuhao Deng1,2, Yi Wang1,3, Xiao-Hua Zhou1,4,5
1Beijing International Center for Mathematical Research, Peking University, 100871 Beijing, China.
Biometrics
|May 14, 2024
概括
本研究引入了两种方法来分析半竞争性风险数据中的治疗效应,分离了对疾病进展和死亡等健康结果的直接和间接影响.
科学领域:
- 生物统计学 生物统计学
- 临床试验 临床试验
- 流行病学 流行病学
背景情况:
- 半竞争性风险发生在一个终端事件 (如死亡) 可以审查一个非终端事件 (如疾病进展),但不是反之.
- 治疗对终端事件的影响可以是直接的或间接的,由非终端事件介导.
研究的目的:
- 在调解分析框架内,提出和评估两种不同的策略,将总治疗效应分解为直接和间接组件.
- 调查在半竞争性风险设置中识别这些分解效应所需的假设.
主要方法:
- 在完全随机的实验中使用调解分析.
- 通过对非终端事件的患病率和危险性进行调整,制定两个分解策略.
- 确定估计的反事实累积发生率和分解的治疗效应的非对称性属性.
主要成果:
- 两种拟议的策略需要不同的假设来确定因果关系的影响.
- 对于反事实累积发病率和分解治疗效应的估计器,确定了对比性属性.
- 模拟研究和真实数据应用表明了这两种分解方法之间的实际差异.
结论:
- 该研究提供了一个强大的框架,以了解在存在半竞争性风险的情况下复杂的治疗效应.
- 提出的方法为研究人员提供了宝贵的工具,用于分析与竞争事件的临床试验数据,增强因果推理.
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