评估单独随机群体治疗试验的分析模型,在嵌套和交叉设计中进行复杂的集群
Jonathan C Moyer1, Fan Li2,3, Andrea J Cook4,5
1Office of Disease Prevention, National Institutes of Health, Bethesda, Maryland, USA.
Statistics in medicine
|September 3, 2024
概括
个人随机组治疗 (IRGT) 试验需要特定的统计模型来计算共享治疗剂. 模拟结果表明,忽视嵌套设计中的多个成员会导致错误,而交叉设计更强大.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 流行病学 流行病学
背景情况:
- 个人随机组治疗 (IRGT) 试验随机选择个体,但使用共享剂来进行治疗.
- 这种共享代理导致参与者之间随机化后的相关性,使分析复杂化.
- 现有的分析模型可能无法充分解决IRGT设计中的复杂集群.
研究的目的:
- 评估复杂集群的IRGT试验的统计模型的性能.
- 在IRGT研究中确定嵌套和交叉代理设计的适当分析模型.
- 在各种集群场景下评估I型错误率.
主要方法:
- 进行了一项模拟研究,以检查候选分析模型.
- 场景包括单个会员,多个会员和单个代理设置.
- 考虑的设计是嵌套和交叉的,具有连续的结果.
主要成果:
- 对于嵌套设计,I型错误率膨胀发生在多个成员不被考虑或模型权重不匹配数据生成时.
- 对交叉设计的分析模型通常保持名义I型错误率.
- 在交叉设计中,每个代理参与者的不平衡是影响错误率的关键因素.
结论:
- 适当的统计建模对于准确分析IRGT试验至关重要.
- 嵌套设计需要仔细考虑多个成员,以避免膨胀的I型错误.
- 交叉设计更强大,但代理不平衡仍然会影响结果.
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