在一个组序列设计中的多个时间到事件终点的家族错误
Henrik F Thomsen1, Nanna L Lausvig2, Christian B Pipper2,3
1Department of Biostatistics, Novo Nordisk A/S, Aalborg, Denmark.
Statistics in medicine
|June 9, 2024
概括
本研究检查了对时间到事件数据的群组顺序设计中的家族错误率 (FWER). 与alpha-spending方法相比,建议采用基于模拟的方法来更好地控制二次终点的FWER.
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
- 生存分析的分析.
背景情况:
- 组序列设计在临床试验中常见,用于早期停止疗效.
- 层次测试用于二级终点来管理多重性.
- 精确控制家族错误率 (FWER) 对于有效的推断至关重要.
研究的目的:
- 通过分层测试,研究在群组顺序设计中对时间到事件终点的全家族错误率 (FWER).
- 在这些设计中识别影响逻辑等级测试统计数据之间的相关性因素.
- 提出一种改进的方法来评估FWER,并为二次终点选择关键值.
主要方法:
- 使用了理论推导和蒙特卡洛模拟.
- 该研究分析了中间和最终分析时的逻辑等级测试统计数据之间的相关性.
- 开发了一种基于模拟的FWER评估方法,并与alpha-spending方法进行了比较.
主要成果:
- 逻辑等级测试统计数据之间的相关性与正常终点的正规相关性不一致.
- 相关性取决于审查水平,终点危险率和危险比率.
- 拟议的基于模拟的方法为二次终点提供了比α支出更好的FWER控制.
结论:
- 对相关性的标准假设可能不适用于组序列试验中的时间到事件数据.
- 在复杂的等级测试场景中,建议采用基于模拟的方法来准确评估FWER和选择关键值.
- 该方法优化了具有多个时间到事件终点的临床试验设计的操作特性.
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