用多种脆弱性和多层次生存模型估计反复事件数据的样本大小
Derek Dinart1,2, Carine Bellera1,2, Virginie Rondeau1,3
1Epicene, University Bordeaux, Inserm, Bordeaux Population Health Research Center, Bordeaux, France.
Journal of biopharmaceutical statistics
|February 9, 2024
概括
在临床研究中,计算复发事件的样本大小至关重要. 这项研究分析了使用脆弱模型对反复发生的时间到事件数据的样本大小,发现样本需求增加,异质性更高.
科学领域:
- 流行病学和临床研究.
- 生物统计学 生物统计学
- 对生存分析的分析.
背景情况:
- 在临床研究中,经常出现住院或骨折等复发性事件.
- 准确的样本大小估计对于反复事件分析的统计能力至关重要.
- 在反复事件数据分析中计算样本大小的现有方法各不相同.
研究的目的:
- 为经常性时间到事件数据提供样本大小要求的深入分析.
- 为了进行样本大小估计,比较不同的脆弱性模型 (共享,层次,联合).
- 提供关于优化研究设计的指导,用于反复事件研究.
主要方法:
- 使用沃尔德型测试统计数据来估计样本大小.
- 研究了从简单的共同脆弱模型到复杂的多层次生存模型.
- 进行模拟以评估不同异质性的样本大小.
- 将该方法应用于AFFIRM-AHF试验数据.
主要成果:
- 样本大小要求随着反复事件数据的异质性增加而增加.
- 包括更多的随访时间较短的患者通常比较少的随访时间较长的患者更有效.
- 不同的脆弱性模型根据研究问题提供了合适的样本大小计算.
结论:
- 脆弱性模型的选择会影响反复事件的样本大小计算.
- 研究设计,平衡患者数量和随访持续时间,对于实现所需事件计数至关重要.
- 本文所介绍的方法为在反复事件研究中确定样本大小提供了一个强大的框架.
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