Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Stat Med. 2021 Feb 28;40(5):1121-1132. doi: 10.1002/sim.8823. Epub 2020 Nov 18.
To ensure that a study can properly address its research aims, the sample size and power must be determined appropriately. Covariate adjustment via regression modeling permits more precise estimation of the effect of a primary variable of interest at the expense of increased complexity in sample size/power calculation. The presence of correlation between the main variable and other covariates, commonly seen in observational studies and non-randomized clinical trials, further complicates this process. Though sample size and power specification methods have been obtained to accommodate specific covariate distributions and models, most existing approaches rely on either simple approximations lacking theoretical support or complex procedures that are difficult to apply at the design stage. The current literature lacks a general, coherent theory applicable to a broader class of regression models and covariate distributions. We introduce succinct formulas for sample size and power determination with the generalized linear, Cox, and Fine-Gray models that account for correlation between a main effect and other covariates. Extensive simulations demonstrate that this method produces studies that are appropriately sized to meet their type I error rate and power specifications, particularly offering accurate sample size/power estimation in the presence of correlated covariates.
为了确保研究能够恰当地解决其研究目标,必须适当地确定样本量和功效。通过回归建模进行协变量调整可以以增加样本量/功效计算的复杂性为代价,更精确地估计主要感兴趣变量的效果。在观察性研究和非随机临床试验中常见的主要变量与其他协变量之间的相关性进一步使这一过程复杂化。尽管已经获得了一些方法来适应特定的协变量分布和模型,但大多数现有的方法要么依赖缺乏理论支持的简单近似值,要么依赖于难以在设计阶段应用的复杂程序。目前的文献缺乏适用于更广泛的回归模型和协变量分布类别的通用、连贯的理论。我们引入了简洁的公式,用于确定具有广义线性、Cox 和 Fine-Gray 模型的样本量和功效,这些模型考虑了主要效应和其他协变量之间的相关性。广泛的模拟表明,这种方法产生的研究能够恰当地满足其Ⅰ型错误率和功效规范,特别是在存在相关协变量的情况下提供准确的样本量/功效估计。