Linn Kristin A, Laber Eric B, Stefanski Leonard A
Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, PA 19104.
Department of Statistics, North Carolina State University, Raleigh, NC 27695.
J Am Stat Assoc. 2017;112(518):638-649. doi: 10.1080/01621459.2016.1155993. Epub 2017 Mar 31.
A dynamic treatment regime is a sequence of decision rules, each of which recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic treatment regimes from data optimize the mean of a response variable. However, the mean may not always be the most appropriate summary of performance. We derive estimators of decision rules for optimizing probabilities and quantiles computed with respect to the response distribution for two-stage, binary treatment settings. This enables estimation of dynamic treatment regimes that optimize the cumulative distribution function of the response at a prespecified point or a prespecified quantile of the response distribution such as the median. The proposed methods perform favorably in simulation experiments. We illustrate our approach with data from a sequentially randomized trial where the primary outcome is remission of depression symptoms.
动态治疗方案是一系列决策规则,每个规则都根据患者病史特征(如既往治疗和治疗结果)推荐治疗方法。现有的从数据中估计最优动态治疗方案的方法会优化响应变量的均值。然而,均值可能并不总是最适合用来总结治疗效果。我们推导了用于优化两阶段二元治疗设置中响应分布的概率和分位数的决策规则估计量。这使得我们能够估计出在预先指定的点上优化响应累积分布函数,或在响应分布的预先指定分位数(如中位数)上优化响应累积分布函数的动态治疗方案。所提出的方法在模拟实验中表现良好。我们用一项序贯随机试验的数据说明了我们的方法,该试验的主要结果是抑郁症状的缓解。