Department of Mathematics and Statistics, Université de Montréal, Montreal, Canada.
Department of Epidemiology, Biostatistics and Occupational Health, McGill University, Montreal, Canada.
Stat Methods Med Res. 2023 May;32(5):868-884. doi: 10.1177/09622802231158733. Epub 2023 Mar 16.
The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the assumption that observation times, that is, treatment and outcome monitoring times, are determined by study investigators. That assumption is often not satisfied in electronic health records data in which the outcome, the observation times, and the treatment mechanism are associated with patients' characteristics. The treatment and observation processes can lead to spurious associations between the treatment of interest and the outcome to be optimized under the dynamic treatment regime if not adequately considered in the analysis. We address these associations by incorporating two inverse weights that are functions of a patient's covariates into dynamic weighted ordinary least squares to develop optimal single stage dynamic treatment regimes, known as individualized treatment rules. We show empirically that our methodology yields consistent, multiply robust estimators. In a cohort of new users of antidepressant drugs from the United Kingdom's Clinical Practice Research Datalink, the proposed method is used to develop an optimal treatment rule that chooses between two antidepressants to optimize a utility function related to the change in body mass index.
医生为治疗慢性疾病而做出的连续治疗决策在统计学文献中被形式化为动态治疗策略。迄今为止,动态治疗策略的方法是在观察时间(即治疗和结果监测时间)由研究调查人员确定的假设下开发的。在电子健康记录数据中,这种假设通常不成立,因为结果、观察时间和治疗机制与患者的特征相关。如果在分析中没有充分考虑到这些因素,治疗和观察过程可能会导致感兴趣的治疗与动态治疗策略下要优化的结果之间产生虚假关联。我们通过将两个逆权重(它们是患者协变量的函数)纳入动态加权最小二乘法中,以开发最优的单阶段动态治疗策略,即个体化治疗规则,来解决这些关联。我们通过实证证明,我们的方法产生了一致的、多重稳健的估计量。在来自英国临床实践研究数据库的抗抑郁药新使用者队列中,我们使用该方法开发了一种最优治疗规则,用于在两种抗抑郁药之间进行选择,以优化与体重指数变化相关的效用函数。