限制平均存活时间和限制平均丧失时间的多组全球测试:可变转换方法
Shuyu Chen1, Mengyao Wang1, Xingyou Zhou1
1Department of Biostatistics, School of Public Health (Guangdong Provincial Key Laboratory of Tropical Disease Research), Southern Medical University, No. 1023, South Shatai Road, Guangzhou, China.
BMC medical research methodology
|May 3, 2025
概括
在多臂临床试验中,新的转换方法提高了使用受限平均存活时间 (RMST) 和受限平均丧失时间 (RMTL) 的治疗效果比较的可靠性,特别是在样本小的情况下.
科学领域:
- 生物统计学 生物统计学
- 临床试验 临床试验
- 生存分析的分析.
背景情况:
- 限制性平均生存时间 (RMST) 和限制性平均丧失时间 (RMTL) 提供了对治疗效果的直观解释.
- 目前的方法主要集中在两组比较上,在小样本中可能不可靠.
- 多臂试验很常见,但对现有的RMST/RMTL分析构成挑战.
研究的目的:
- 开发一个全球测试框架,用于RMST和RMTL的多组比较.
- 为了解决在小样本大小和高审查率的非对称方法的局限性.
- 在复杂的试验设计中提高治疗效果比较的可靠性和可解释性.
主要方法:
- 提出了一个使用变量转换方法 (log,clog-log,arcsine平方根,logit) 的全球测试框架.
- 扩展了框架,以处理RMTL的竞争风险场景.
- 使用蒙特卡洛模拟来评估I型错误和功率的评估性能.
主要成果:
- 转换RMST和RMTL全球测试有效控制了小样本和高审查的I型错误.
- 与未经转换的方法相比,这些方法显示了更好的统计能力.
- 建议进行特定的转换:基于事件频率的RMST,arcsine平方根,RMTL的logit或clog-log.
结论:
- 基于转换的全球测试为多组治疗效果的比较提供了一个灵活的,无假设的工具.
- 这些方法提高了临床试验分析的可靠性和解释性.
- 一个R包"compRM"可用于实现这些新的统计方法.
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