Wang Jijia, Zhang Song, Ahn Chul
Department of Statistical Science, Southern Methodist University, Dallas, TX.
Department of Clinical Sciences, UT Southwestern Medical Center, Dallas, TX.
Commun Stat Theory Methods. 2020;49(1):116-124. doi: 10.1080/03610926.2018.1532004. Epub 2018 Dec 21.
In many cluster randomization studies, cluster sizes are not fixed and may be highly variable. For those studies, sample size estimation assuming a constant cluster size may lead to under-powered studies. Sample size formulas have been developed to incorporate the variability in cluster size for clinical trials with continuous and binary outcomes. Count outcomes frequently occur in cluster randomized studies. In this paper, we derive a closed-form sample size formula for count outcomes accounting for the variability in cluster size. We compare the performance of the proposed method with the average cluster size method through simulation. The simulation study shows that the proposed method has a better performance with empirical powers and type I errors closer to the nominal levels.
在许多整群随机试验中,群的大小并非固定不变,可能差异很大。对于这些试验,假设群大小恒定来估计样本量可能会导致效能不足的试验。已开发出样本量公式,以纳入具有连续和二元结局的临床试验中群大小的变异性。计数结局在整群随机试验中经常出现。在本文中,我们推导了一个考虑群大小变异性的计数结局的封闭式样本量公式。我们通过模拟比较了所提出方法与平均群大小法的性能。模拟研究表明,所提出的方法具有更好的性能,其经验效能和I型错误更接近名义水平。