在RCT中因死亡而缩短的结果:对幸存者的平均因果效应的模拟研究
Stefanie von Felten1, Chiara Vanetta1,2, Christoph M Rüegger3
1Department of Biostatistics at Epidemiology, Biostatistics and Prevention Institute, University of Zurich, Zurich, Switzerland.
Biometrical journal. Biometrische Zeitschrift
|June 12, 2025
概括
估计因死亡而缺失的治疗结果的治疗效果是具有挑战性的. 幸存者平均因果效应 (SACE) 和多重归算方法可以比随机对照试验中的完整病例分析更好地减少偏差.
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:
- 在随机对照试验 (RCT) 中估计无偏见的治疗效应,由于死亡截止的连续结果使其复杂化.
- 幸存者平均因果效应 (SACE) 是一种方法,但依赖于无法测试的假设.
研究的目的:
- 为了比较SACE估计的性能与完整病例分析 (CCA) 和多重归算 (MI) 在死亡的情况下连续结果.
- 根据各种治疗效果场景对结果和生存情况来评估这些方法.
主要方法:
- 进行了一项模拟研究,采用九种情景,对治疗对认知发育和2年生存时间的影响有所不同.
- 比较偏差,平均平方误差,SACE,CCA和MI估计器与真实治疗效应和SACE的覆盖范围.
主要成果:
- 与CCA相比,SACE和MI提供了类似的治疗效果估计,大大减少了偏差.
- 无论是SACE还是MI,都表现出对共变量遗漏的稳定性,这表明对假设违规的弹性.
结论:
- 在RCT中,SACE和MI是处理因死亡而缩短的连续结果的有价值的方法,特别是当死亡率与人群固有时.
- 虽然SACE假设可能被违反,但这些方法在特定的临床试验环境中比CCA具有实际优势.
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