基于排名的方法来评估等价性/非劣等性与测定灵敏度在一个三臂试验与顺序终点三臂试验
Shi-Fang Qiu1, Dai-Min Li1, Wai-Yin Poon2
1Department of Statistics and Data Science, Chongqing University of Technology, Chongqing, China.
Statistical methods in medical research
|February 12, 2026
概括
本研究介绍了在有序结果的三臂试验中评估治疗等价性的胜利概率. 提出的方法为顺序数据提供可靠的分析,改善临床试验设计.
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
- 顺序数据分析 顺序数据分析
背景情况:
- 现有的三臂试验中测定灵敏度的同等性/非劣等性方法对于顺序终点是有限的.
- 顺序数据缺乏指标性质,使连续数据的标准分析方法具有潜在的错误性.
- 获胜概率为比较研究中的治疗效果量化提供了可靠的衡量标准.
研究的目的:
- 在三臂试验中使用获胜概率来评估测试灵敏度的等价性/非劣质性.
- 开发和评估适用于临床试验中的顺序终点的统计方法.
- 为确定样本大小提供指导,以实现所需的统计能力.
主要方法:
- 在同步置信区间 (SCI) 中利用获胜概率进行等效/非劣等性评估.
- 应用对参数假设稳定的方法,适用于顺序和连续结果.
- 调查基于费舍尔-z转换的SCI,方差估计恢复SCI与逻辑转换,以及逻辑与弧形转换的置信限.
主要成果:
- 经验结果表明,提出的方法 (费舍尔-z,logit转换) 显示出良好的性能.
- 覆盖概率接近于名义信任水平,表明可靠性.
- 研究了样本大小确定方法,与假设测试-间隔估计二元性保持一致.
结论:
- 拟议的基于胜率的方法提供了一个有效的方法,用于评估三臂试验中的测定灵敏度的等价性/非劣等性,特别是对于顺序数据.
- 这些方法灵活,适用于顺序和连续结果,没有严格的参数假设.
- 该研究为临床试验设计和分析提供了实用工具,以手术后恶心和吐预防示例为例.
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