使用GEE型模型分析零膨胀的聚类纵向有序结局：以牙釉质氟中毒研究为例
Analyzing zero-inflated clustered longitudinal ordinal outcomes using GEE-type models with an application to dental fluorosis studies
AI总结：
本研究基于爱荷华氟化物研究数据，提出一种结合广义估计方程和James-Stein收缩的统一建模框架，用于分析零膨胀聚类纵向有序结局，并识别出早期氟暴露与牙釉质氟中毒的年龄特异性风险因素。
摘要：
受爱荷华氟化物研究（IFS）的启发，该研究追踪了从儿童期到青年期（年龄9、13、17和23岁）的氟化物摄入和牙齿结局，我们分析了牙釉质氟中毒——一种由牙釉质形成期间过量氟化物暴露引起的疾病。在此背景下，牙齿表面的氟中毒评分表现为零膨胀、聚类和纵向有序结局，促使我们开发一个统一的建模框架。利用广义估计方程（GEEs），我们为氟中毒的存在和严重程度分别构建模型，并提出一个通过共享协变量连接这些组件的组合模型。为了提高估计效率并在时间点之间借用强度，我们引入了James-Stein收缩估计量。我们比较了几种工作相关结构，包括一种数据驱动的jackknife结构，并通过秩聚合进行模型选择。模拟研究验证了所提出模型的有限样本性能，基于自助法的功效分析进一步确认了检验程序的有效性。在对IFS数据的分析中，早期生活中每日总氟化物摄入量、家庭平均自来水氟化物浓度以及特定牙齿和区域被确定为牙釉质氟中毒的重要风险因素。上颌侧切牙和更靠近牙龈的区域在不同年龄显示出保护效应。这些发现揭示了早期生活暴露与牙釉质氟中毒在青年早期进展之间新颖的年龄特异性关联。
英文摘要：
Motivated by the Iowa Fluoride Study (IFS), which tracked fluoride intake and dental outcomes from childhood to young adulthood (ages 9, 13, 17, and 23), we analyze dental fluorosis - a condition caused by excessive fluoride exposure during enamel formation. In this context, fluorosis scores across tooth surfaces present as zero-inflated, clustered, and longitudinal ordinal outcomes, prompting the development of a unified modeling framework. Leveraging generalized estimating equations (GEEs), we construct separate models for the presence and severity of fluorosis and propose a combined model that links these components though shared covariates. To improve estimation efficiency and borrowing strength across timepoints, we incorporate James-Stein shrinkage estimators. We compare several working correlation structures, including a data-driven jackknifed structure, and perform model selection via rank aggregation. Simulation studies validate the finite-sample performance of the proposed models, and a bootstrap-based power analysis further confirms the validity of the testing procedure. In our analysis of the IFS data, early-life total daily fluoride intake, average home water fluoride concentration, and specific teeth and zones emerge as significant risk factors for dental fluorosis. Maxillary lateral incisors and zones closer to the gum show protective effects across different ages. These findings reveal novel age-specific associations between early-life exposures and the progression of dental fluorosis through early adulthood.
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