在小样本交叉试验中如何分析连续和离散的重复测量?
Johan Verbeeck1, Martin Geroldinger2,3, Konstantin Thiel2,3
1Data Science Institute (DSI), Interuniversity Institute for Biostatistics and statistical Bioinformatics (I-BioStat), Hasselt University, Hasselt, Belgium.
Biometrics
|August 17, 2023
概括
分析罕见疾病试验的重复测量和交叉设计需要谨慎的统计方法. 这项研究建议基于结果类型和治疗效果持续时间的特定分析方法,以实现最佳的数据利用.
科学领域:
- 生物统计学 生物统计学
- 临床试验 临床试验
- 罕见疾病 罕见疾病
背景情况:
- 重复措施交叉设计为小样本罕见疾病试验提供了优势.
- 在这些试验中分析受试者内部和治疗期间内聚类的数据存在挑战.
研究的目的:
- 为了比较分析重复测量的统计方法,在小样本罕见疾病试验中进行交叉设计.
- 根据结果类型和治疗效果持续时间,为最佳数据分析提供建议.
主要方法:
- 一个真实数据模拟研究,基于一个表皮溶解牛简单试验.
- 非参数边缘模型的比较,一般化的对比模型,GEE型模型和参数模型的平均值.
- 对重复的二进制和计数数据进行分析.
主要成果:
- 选择方法取决于结果类型和治疗影响的时间点的数量.
- 非参数边缘模型适用于检测纵向轮形状的差异.
- 对于单个时间点处理效应的二进制结果,建议进行参数模型平均化;否则,建议进行通用的对比比较.
结论:
- 建议使用特定的统计方法来分析罕见疾病试验,重复测量交叉设计.
- 一般对对比和参数模型平均提供可解释的效果大小,并有效处理不完整的数据.
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