单臂临床试验的样本大小考虑与使用马分布的时间到事件终点的单臂临床试验
Junqiang Dai1, Jianghua He1, Milind A Phadnis1
1Department of Biostatistics and Data Science, University of Kansas Medical Center, Kansas City, KS, USA.
Contemporary clinical trials communications
|September 4, 2024
概括
本研究介绍了用于计算单臂临床试验中样本大小的玛分布,该试验具有时间到事件终点. 这为现有方法提供了更灵活的替代方案,当疾病模式偏离标准假设时,提高了准确性.
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:
- 单臂临床试验经常使用时间到事件 (TTE) 终点.
- 当前的统计软件在这些试验中对样本大小计算的选择有限.
- 通常使用的参数模型假定生存时间的指数或韦布尔分布.
研究的目的:
- 建议用玛分布作为一个灵活的参数选项来计算单臂试验中的样本大小,使用TTE终点.
- 从现有的文献中开发一种估计玛形状参数的方法.
- 评估这一估计的准确性和分配错误规格的影响.
主要方法:
- 概述了一种使用已知形状参数的马分布的样本大小计算方法.
- 详细介绍了从已发表的数据中提取玛形状估计方法 (中位数,IQR).
- 进行了模拟,以评估提取的参数的准确性和错误规格的影响.
主要成果:
- 可以从小样本大小 (<60) 和低审查率 (<20%) 的研究中获得合理准确的玛形状估计.
- 使用这些估计可以设计新的单臂研究.
- 对真实生存时间分布的错误说明可能导致对样本大小的低估或高估.
结论:
- 马分布为单臂TTE试验设计提供了对指数和韦布尔分布的有价值的替代方案.
- 基于模拟的评估证实了马形状估计的可靠性.
- 建议小心谨慎,以防止对基本生存时间分布的错误规范.
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