Ten Have T R, Kunselman A R, Tran L
Department of Biostatistics and Clinical Epidemiology, University of Pennsylvania College of Medicine, Philadelphia 19104-6024, USA.
Stat Med. 1999 Apr 30;18(8):947-60. doi: 10.1002/(sici)1097-0258(19990430)18:8<947::aid-sim95>3.0.co;2-b.
We compare mixed effects logistic regression models for binary response data with two nested levels of clustering. The comparison of these models occurs in the context of developmental toxicity data sets, for which multiple types of outcomes (first level) are measured on each rat pup (second level) nested within a litter (third level). Because the nested nature of such data is occasionally accommodated by ignoring one level of clustering, we consider three models: (i) a three-level model adjusting for clustering due to both pup and litter (M1); (ii) a two-level model adjusting for just pup (M2); and (iii) another two-level model adjusting for just litter (M3). The three types of effects of interest are: (i) differences among malformation types (first-level effects); (ii) differences among groups of pups (for example, sex of pup, second-level effects); and (iii) differences among groups of litters (for example, dose, third-level effects). Simulations and data analyses suggest that the M3 model leads to more bias than the M1 or M2 models for all three types of effects. In terms of coverage of confidence intervals for fixed effects log odds ratio parameters, the M1 model achieves nominal coverage, whereas the M2 model reduces coverage for the third-level effects and the M3 model obtains poor coverage for both first- and second-level effects. These reductions in coverage for certain model-parameter combinations worsen as baseline risk increases. The data analyses support these simulation-based conclusions to some extent.
我们比较了用于二元响应数据且具有两个嵌套聚类水平的混合效应逻辑回归模型。这些模型的比较是在发育毒性数据集的背景下进行的,对于该数据集,在窝(第三级)内嵌套的每只幼鼠(第二级)上测量了多种类型的结果(第一级)。由于此类数据的嵌套性质有时会通过忽略一个聚类水平来处理,我们考虑三种模型:(i)一个三级模型,对幼鼠和窝引起的聚类进行调整(M1);(ii)一个二级模型,仅对幼鼠进行调整(M2);以及(iii)另一个二级模型,仅对窝进行调整(M3)。感兴趣的三种效应类型为:(i)畸形类型之间的差异(第一级效应);(ii)幼鼠组之间的差异(例如,幼鼠的性别,第二级效应);以及(iii)窝组之间的差异(例如,剂量,第三级效应)。模拟和数据分析表明,对于所有三种效应类型,M3模型比M1或M2模型导致更多偏差。就固定效应对数比值比参数的置信区间覆盖范围而言,M1模型达到了名义覆盖范围,而M2模型降低了第三级效应的覆盖范围,M3模型对第一级和第二级效应的覆盖范围都很差。随着基线风险增加,某些模型参数组合的覆盖范围减少情况会恶化。数据分析在一定程度上支持了这些基于模拟的结论。