在阶段集群随机试验中的预期效应上
Hao Wang1,2, Xinyuan Chen3, Katherine R Courtright4,5
1Department of Biostatistics, Yale University, New Haven, Connecticut, USA.
Statistics in medicine
|February 10, 2026
概括
在阶段集群随机试验 (SW-CRTs) 中忽视预期效应可能会导致治疗效应估计偏差. 本研究引入了一个框架,以解决SW-CRT中的预测问题,提高这些复杂试验的准确性和样本大小计算.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 流行病学 流行病学
背景情况:
- 逐步集群随机试验 (SW-CRTs) 越来越多地用于评估现实世界的干预措施.
- 对SW-CRT的标准分析假定没有预测干预,当意识到推出时间影响行为时,可能会受到侵犯.
- 预期效应可能导致SW-CRT中偏见的治疗效应估计.
研究的目的:
- 开发一个分析框架来解决和量化SW-CRT中的预测效应.
- 评估忽视预期对治疗效果估计器的影响.
- 提供更新的样本大小公式,考虑预期和暴露时间异质性.
主要方法:
- 使用一系列线性混合模型推导估计者的预期.
- 开发分析框架,以纳入预测效应.
- 模拟研究和经验分析,以比较预期调整和没有预期调整的估计.
主要成果:
- 忽视预期效应会导致在SW-CRT中偏差治疗效应估计.
- 拟议的框架提供了调整后的估计,以减轻偏差.
- 更新的样本大小公式证明了预测对研究功率的影响.
结论:
- 预期效应的计算对于SW-CRT中准确的治疗效应估计至关重要.
- 开发的框架和更新的样本大小计算提高了SW-CRT的方法严格性.
- 提供了实施预期调整分析的实际考虑.
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