范围审查确定了在集群随机试验中定义估值的额外考虑因素
Dongquan Bi1, Andrew Copas1, Fan Li2
1MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, University College London, London, UK.
Journal of clinical epidemiology
|October 12, 2025
概括
定义集群随机试验 (CRT) 的估计值需要考虑标准指南之外的八个额外项目. 这些因素在复杂的CRT设计中为临床医生,患者和决策者确保了治疗效果的明确性.
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:
- 估计定义了研究量化的特定治疗效应,对于可解释的结果至关重要.
- 国际协调理事会附录E9(R1) 提供了估计和定义的五个属性,主要用于单独随机试验.
- 集群随机试验 (CRT) 在估计和定义方面引入了独特的复杂性,这是由于组级随机化.
研究的目的:
- 确定具体在集群随机试验 (CRT) 的背景下定义估计值的额外考虑因素.
- 为了补充现有的ICH E9 (((R1) 准则,以CRT特定的因子来进行可靠的估计和规范.
主要方法:
- 在多个数据库和个人图书馆进行系统的文献搜索.
- 识别详细介绍估计和定义方面,但未被ICH E9 (R1) 附录所涵盖的CRT的文章.
- 将调查结果综合到CRT估计和考虑的额外项目列表中.
主要成果:
- 从46个符合条件的文章中确定了8个关键项目,用于CRT中定义估值.
- 这些包括:集群的种群,个体的选择偏差,暴露时间,权重方法 (个体平均值与集群平均值),边际与集群特定措施,处理集群级间流动事件,干扰/溢出,以及集群之间的参与者消耗/移动.
- 这些项目解决的复杂性超出了通常在个别随机试验中遇到的复杂性.
结论:
- 本综述强调了在CRT中估计和定义的重要额外因素.
- 进行CRT的调查人员应该纳入这八个项目,以确保估计是明确和相关的.
- 清晰的估计增强了CRT发现对临床医生,患者和决策者等最终用户的实用性.
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