Department of Statistics, University of Michigan, Ann Arbor, MI 48109-1107, USA.
Stat Methods Med Res. 2010 Jun;19(3):317-43. doi: 10.1177/0962280209105013. Epub 2009 Jul 16.
A dynamic treatment regime is a set of decision rules, one per stage, each taking a patient's treatment and covariate history as input, and outputting a recommended treatment. In the estimation of the optimal dynamic treatment regime from longitudinal data, the treatment effect parameters at any stage prior to the last can be non-regular under certain distributions of the data. This results in biased estimates and invalid confidence intervals for the treatment effect parameters. In this article, we discuss both the problem of non-regularity, and available estimation methods. We provide an extensive simulation study to compare the estimators in terms of their ability to lead to valid confidence intervals under a variety of non-regular scenarios. Analysis of a data set from a smoking cessation trial is provided as an illustration.
动态治疗方案是一组决策规则,每个阶段一个规则,每个规则都以患者的治疗和协变量历史记录作为输入,并输出推荐的治疗方案。在从纵向数据中估计最优动态治疗方案时,在最后一个阶段之前的任何阶段,在数据的某些分布下,治疗效果参数可能是非正则的。这会导致治疗效果参数的估计值有偏差,置信区间无效。本文讨论了不规则性问题以及可用的估计方法。我们进行了广泛的模拟研究,比较了在各种不规则情况下能够产生有效置信区间的估计量。提供了一个来自戒烟试验的数据集的分析作为说明。