设计和分析个别随机群体治疗试验的设计和分析,包括时间到事件的结果
1Department of Biostatistics and Bioinformatics, Duke University, Durham, NC, 27705, USA. sinho.jung@duke.edu.
Lifetime data analysis
|June 3, 2025
概括
本研究引入了修改后的日志等级测试和样本大小公式,用于具有时间到事件结果的个人随机组治疗 (IRGT) 试验. 这些方法准确地确定样本大小,用于在复杂的群组随机设计中比较生存数据.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 生存分析的分析.
背景情况:
- 个人随机组治疗 (IRGT) 试验涉及个体随机化,但在实验组中分配组级治疗.
- 由于共同的特征,治疗组内的结果可能是依赖的,这使标准的统计分析复杂化.
- 时间到事件的结果在临床研究中很常见,需要专门的统计方法.
研究的目的:
- 为IRGT试验开发一个统计测试和样本大小公式,具有时间到事件结果.
- 解决实验手臂中集群内结果的依赖性.
- 在IRGT试验中提供准确样本大小计算和最佳研究设计的方法.
主要方法:
- 修改了对IRGT试验生存数据的两个样本日志等级测试.
- 采样大小公式的推导,包括边际和双变生存分布,集群大小和积累参数.
- 开发最佳设计以尽量减少样本大小或成本.
主要成果:
- 拟议的修改后的日志排名测试统计数据有效控制了I型错误率.
- 导出的样本大小公式为IRGT试验提供了准确的功率.
- 模拟和真实数据示例验证了拟议的方法.
结论:
- 开发的统计方法适合在IRGT试验中分析时间到事件数据.
- 样本大小公式有助于高效准确地规划这些复杂的临床试验.
- 最佳的设计策略可以进一步提高IRGT试验的效率.
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