包括未曝光的集群提高了稳定效应分析的精度,分阶集群随机试验的二进制和计数结果
Kenneth Menglin Lee1,2, Grace Meijuan Yang3,4, Yin Bun Cheung5,6,7
1Centre for Quantitative Medicine, Duke-NUS Medical School, Singapore, 169857, Singapore. klee@u.duke.nus.edu.
BMC medical research methodology
|October 29, 2024
概括
将未暴露的集群纳入步集群随机试验 (SW-CRT) 的固定效应分析中,具有二进制或计数结果,可以提高估计器的精度和功率. 这一发现增强了SW-CRT的分析,即使有早期终止或安全问题.
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:
- 固定效应模型提供了一个混合效应模型的替代方案,用于步集群随机试验 (SW-CRT),控制混因子,并保持一些集群的I型错误控制.
- SW-CRT通常涉及所有集群交叉进行干预,但由于早期终止或安全问题,一些集群可能不会暴露.
- 之前的研究表明,未暴露的集群在固定效应的SW-CRT分析中提高了连续结果的精度;对非连续结果的扩展是未知的.
研究的目的:
- 通过数学证明,包括未暴露的集群可以提高固定效应干预效应估计器对具有二进制和计数结果的SW-CRT的精度.
- 探索将未暴露的集群纳入模拟和真实世界SW-CRT数据中的实用好处,并提供二进制或计数结果.
主要方法:
- 数学推导来证明包括未暴露的集群在固定效应SW-CRT分析对二进制和计数结果的理论好处.
- 使用二进制和计数结果数据进行模拟研究,以评估未暴露的集群包含对估计器精度,功率和根平均平方误差 (RMSE) 的影响.
- 对现实世界的息护理干预SW-CRT与二进制结果进行分析,以评估对标准错误和置信区间的实际影响.
主要成果:
- 模拟证实,结合未暴露的集群显著提高了干预效应估计器的精度,功率,并降低了干预效应估计器的根平均平方误差 (RMSE).
- 将未暴露的集群纳入具有二元结果的息护理SW-CRT中,导致标准误差更小,Wald 95%信心区间更窄.
- 这些发现支持从连续到二进制和计数结果SW-CRT分析的好处的概括性.
结论:
- 在固定效应分析中包含未暴露的集群显著提高了SW-CRT干预效应估计器的精度,功率和RMSE,具有二进制或计数结果.
- 这种方法可以更准确,更有效地估计SW-CRT中的干预效应,特别是当一些集群没有接受干预时.
- 该研究证实,未暴露的集群对于改善各种结果类型中固定效应SW-CRT分析的统计效率是有价值的.
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