对随机试验的不同人群层次总结措施与时间到事件的结果进行比较,重点是非劣势试验
Matteo Quartagno1, Tim P Morris1, Duncan C Gilbert1
1MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK.
Clinical trials (London, England)
|June 20, 2023
概括
这项研究比较了临床试验的时间到事件总结措施,发现限制平均存活时间 (RMST) 的差异往往是最强大的,特别是假设比例危险时. 这一发现有助于为非劣等性试验选择最佳的统计方法.
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:
- 在临床试验中,特别是非劣势试验中,对临床试验的结果至关重要.
- 不同的总结措施意味着不同的虚假假设,影响试验设计和解释.
- 危险比率是常用的,但限制平均存活时间 (RMST) 的差异可能在某些场景中提供更大的功率.
研究的目的:
- 为了比较三个常见的总结指标的性能:危险比率,RMST的差异和固定时间点的生存差异.
- 通过使用这些总结措施,调查假设比例危险对试验运行特征的影响.
- 为非劣势试验选择适当的总结措施提供指导,并提供时间到事件的结果.
主要方法:
- 在比例危险框架内进行了模拟研究.
- 在RMST的差异和生存的差异被估计非参数 (没有比例危险假设).
- 这三项测量均使用比例危险假设的灵活生存回归进行参数估计.
主要成果:
- 总结措施的相对表现取决于在比较危险比率 (假设比例危险) 与其他情况 (不假设比例危险) 时的情景而有很大差异.
- 与非参数方法相比,假设比例危险始终产生了相当大的功率增长.
- 在使用灵活参数回归来估计比例危险时,RMST的差异通常是最强大的总结措施.
结论:
- 在分析中反映近似比例危险可以显著增加RMST差异和生存差异的功率.
- 摘要措施的选择主要应基于临床相关性.
- 在临床上有理由时,在比例危险下估计的RMST的差异通常与最强大的统计测试有关.
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