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审查当前在设计和分析极小的步骤集群随机试验的现实实践
Guangyu Tong1,2,3, Pascale Nevins4,5, Mary Ryan6,7
1Section of Cardiovascular Medicine, Department of Internal Medicine, Yale School of Medicine, New Haven, CT, USA.
步骤集群随机试验中只有少数集群是常见的,但往往缺乏适当的统计分析. 设计和分析这些小型试验的最佳实践尚未得到广泛采用,需要进一步研究.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 步集群随机试验 (SW-CRT) 提供了效率,但缺乏对最小集群数量的指导.
- 小型SW-CRT普遍存在,但理由和分析方法往往不足.
- 在SW-CRT中的纵向相关结构需要仔细考虑,以避免低估样本大小.
研究的目的:
- 从2016年到2022年,审查已发表的小型SW-CRT (2-6个集群).
- 了解在SW-CRT中使用少量集群的原因.
- 描述小型SW-CRT中使用的设计和分析方法.
主要方法:
- 系统的电子搜索确定了2016-2022年间出版的SW-CRT.
- 选择了一组试验,随机选择了两到六个集群.
- 数据由两个独立的审稿人提取,意见分歧通过讨论解决.
主要成果:
- 确定了61个小型SW-CRT,平均共有1426名参与者.
- 小集群数量的原因包括尽量减少污染,集群可用性和物流.
- 大多数试验 (83.6%) 报告了样本大小计算,但只有一个解释了明显的集群内相关性.
- 很少有试验 (6.6%) 使用小样本校正或适用于小集群的适当统计方法.
结论:
- 目前设计和分析小型SW-CRT的实践不足.
- 标准样本大小方法可能会产生误导性的低集群要求.
- 对于小型SW-CRT,推使用GEE,GLMM与小样本校正,贝叶斯分析或顺序测试等先进方法.
- 进一步的研究对于建立小型SW-CRT的最佳实践至关重要.
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