Sample size estimation for the ratio of count outcomes in a cluster randomized trial using GEE.

IF 0.8 4区 数学 Q4 STATISTICS & PROBABILITY
Jijia Wang, Song Zhang, Chul Ahn
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引用次数: 0

Abstract

Count outcomes often occur in cluster randomized trials. Particularly in the context of epidemiology, the ratio of incidence rates has been used to assess the effectiveness of an intervention. In practice, cluster sizes typically vary across clusters, and sample size estimation based on a constant cluster size assumption may lead to underpowered studies. To address this issue, we propose a sample size method based on the generalized estimating equation (GEE) approach to test the ratio of two incidence rates. A closed-form sample size formula is presented, which is flexible to account for unbalanced randomization and randomly varying cluster sizes. Simulations were performed to assess its performance. In cluster randomized trials of vaccine efficacy, the ratio of disease incidence rates has been frequently used to demonstrate that the vaccine reduces the occurrence of a disease compared to placebo or active control. An application example to the design of a vaccine efficacy cluster randomized trial is presented.

使用GEE的聚类随机试验中计数结果比率的样本量估计。
计数结果经常出现在聚类随机试验中。特别是在流行病学的背景下,发病率比率已被用来评估干预措施的有效性。在实践中,集群大小通常因集群而异,基于恒定集群大小假设的样本大小估计可能会导致研究效果不足。为了解决这一问题,我们提出了一种基于广义估计方程(GEE)方法的样本量方法来检验两种发病率的比率。提出了一个封闭的样本大小公式,该公式可以灵活地考虑不平衡随机化和随机变化的簇大小。通过仿真对其性能进行了评估。在疫苗功效的群随机试验中,经常使用疾病发病率的比率来证明,与安慰剂或主动对照相比,疫苗减少了疾病的发生。给出了一个应用于疫苗功效群随机试验设计的实例。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
2.00
自引率
12.50%
发文量
320
审稿时长
7.5 months
期刊介绍: The Theory and Methods series intends to publish papers that make theoretical and methodological advances in Probability and Statistics. New applications of statistical and probabilistic methods will also be considered for publication. In addition, special issues dedicated to a specific topic of current interest will also be published in this series periodically, providing an exhaustive and up-to-date review of that topic to the readership.
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