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Dynamic treatment regimes: technical challenges and applications

机译:动态治疗方案:技术挑战和应用

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摘要

Dynamic treatment regimes are of growing interest across the clinical sciences because these regimes provide one way to operationalize and thus inform sequential personalized clinical decision making. Formally, a dynamic treatment regime is a sequence of decision rules, one per stage of clinical intervention. Each decision rule maps up-to-date patient information to a recommended treatment. We briefly review a variety of approaches for using data to construct the decision rules. We then review a critical inferential challenge that results from nonregularity, which often arises in this area. In particular, nonregularity arises in inference for parameters in the optimal dynamic treatment regime; the asymptotic, limiting, distribution of estimators are sensitive to local perturbations. We propose and evaluate a locally consistent Adaptive Confidence Interval (ACI) for the parameters of the optimal dynamic treatment regime. We use data from the Adaptive Pharmacological and Behavioral Treatments for Children with ADHD Trial as an illustrative example. We conclude by highlighting and discussing emerging theoretical problems in this area.
机译:动态治疗方案在整个临床科学中都越来越引起人们的兴趣,因为这些方案提供了一种操作方法,可以为后续的个性化临床决策提供依据。正式地,动态治疗方案是一系列决策规则,每个阶段的临床干预。每个决策规则都会将最新的患者信息映射到推荐的治疗方法。我们简要回顾了使用数据构建决策规则的多种方法。然后,我们回顾由非规律性导致的关键推理推断挑战,这种挑战通常在这一领域出现。特别是,在最佳动态治疗方案中,对参数的推论出现了不规则性。估计的渐近,极限分布对局部扰动敏感。我们提出并评估最佳动态治疗方案参数的局部一致的自适应置信区间(ACI)。我们以儿童多动症试验的适应性药理和行为治疗数据为例。最后,我们重点介绍并讨论了该领域出现的理论问题。

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