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Sample Size Calculation for Comparing Time-Averaged Responses in K-Group Repeated-Measurement Studies

机译:样本量计算时间平均响应在K-组重复测量比较研究

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摘要

Many clinical trials compare the efficacy of K (≥3) treatments in repeated measurement studies. However, the design of such trials have received relatively less attention from researchers. derived a closed-form sample size formula for two-sample comparisons of time-averaged responses using the generalized estimating equation (GEE) approach, which takes into account different correlation structures and missing data patterns. In this paper, we extend the sample size formula to scenarios where K (≥3) treatments are compared simultaneously to detect time-averaged differences in treatment effect. A closed-form sample size formula based on the noncentral χ2 test statistic is derived. We conduct simulation studies to assess the performance of the proposed sample size formula under various correlation structures from a damped exponential family, random and monotone missing patterns, and different observation probabilities. Simulation studies show that empirical powers and type I errors are close to their nominal levels. The proposed sample size formula is illustrated using a real clinical trial example.
机译:许多临床试验在重复测量研究中比较了K(≥3)治疗的疗效。但是,此类试验的设计受到研究人员的关注相对较少。推导了使用广义估计方程(GEE)方法进行时间平均响应的两个样本比较的封闭形式样本大小公式,该公式考虑了不同的相关结构和缺失的数据模式。在本文中,我们将样本量公式扩展到同时比较K(≥3)种治疗方案以检测治疗效果的时间平均差异的方案。推导了基于非中心χ 2 检验统计量的封闭样本大小公式。我们进行了仿真研究,以评估来自阻尼指数族,随机和单调缺失模式以及不同观察概率的各种相关结构下拟议样本量公式的性能。仿真研究表明,经验功效和I类错误均接近其标称水平。建议的样本量公式使用一个实际的临床试验示例进行了说明。

著录项

  • 期刊名称 other
  • 作者

    Song Zhang; Chul Ahn;

  • 作者单位
  • 年(卷),期 -1(58),-1
  • 年度 -1
  • 页码 283–291
  • 总页数 17
  • 原文格式 PDF
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