通过联合建模分析对象阶段子集群随机试验的分析,具有不可忽视的脱学率
Alessandro Gasparini1,2, Michael J Crowther1, Emiel O Hoogendijk3
1Red Door Analytics AB, Stockholm, Sweden.
Statistics in medicine
|February 18, 2025
概括
这项研究引入了一种联合纵向生存模型,以准确分析阶段形集群随机试验 (CRT) 与信息丢失,这对于死亡率研究至关重要. 该方法通过考虑缺失的数据来改善估计,提高CRT发现的可靠性.
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
- 纵向数据分析 纵向数据分析
背景情况:
- 阶段结集群随机试验 (CRT) 涉及复杂的分析由于集群内相关性和信息性脱落,特别是在高死亡率队列.
- 死亡率的缺失结果可能会导致治疗效果估计的偏差,并降低闭合队列CRT中的统计能力.
- 在纵向研究中,现有的方法可能无法充分解决因死亡而导致的不可忽视的缺失结果.
研究的目的:
- 扩展线性混合效应模型,以分析具有信息性脱落的闭合队列级别形CRT.
- 开发一个联合纵向生存建模框架,明确建模脱学过程.
- 通过蒙特卡洛模拟来评估拟议方法的性能.
主要方法:
- 开发了联合纵向生存模型,将一个时间到事件的脱学子模型与一个纵向结果子模型联系起来.
- 该方法从线性混合效应模型扩展,以适应信息缺失.
- 对于纵向组件,考虑了持续干预和一般的时间对治疗效果参数化.
主要成果:
- 拟议的联合建模方法有效地适应了阶段形CRT中的信息丢失.
- 蒙特卡洛模拟在各种数据生成场景下证明了该方法的性能.
- 重新分析脆弱的老年人:护理过渡期 (ACT) 试验说明了实际应用.
结论:
- 联合纵向生存模型提供了一个强大的框架,用于分析具有信息性脱落的阶段式CRT.
- 这种方法对于在高死亡率的研究中对治疗效果的公正估计至关重要.
- 该方法提高了复杂的纵向试验设计结果的有效性和力量.
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