Local control for identifying subgroups of interest in observational research: persistence of treatment for major depressive disorder
Local control for identifying subgroups of interest in observational research: persistence of treatment for major depressive disorder
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DOI：
10.1002/mpr.1390
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发表时间：
2013-09-01
影响因子：
3.1
通讯作者：
Obenchain, Robert L.
中科院分区：
文献类型：
--
作者：
Faries, Douglas E.;Chen, Yi;Obenchain, Robert L.
护理人员经常面临相互竞争的治疗方案之间的抉择。由于其样本量、不同的人群和现实世界的环境，大型观察性医疗保健数据库为研究患者反应的异质性以指导护理人员的决策提供了绝佳的机会。局部对照是一种很有前途的工具，可使用观察数据来检测对一种治疗相对于另一种治疗具有差异反应的患者亚组。虽然标准数据挖掘方法可以找到对特定人群具有最佳反应的亚组，但检测揭示治疗差异的亚组，同时调整观察数据中的混杂因素是具有挑战性的。局部控制利用无监督聚类来形成非参数患者水平的反事实治疗差异，并将其显示为效应大小估计的观察到的分布。然后，分类和回归树 (CART) 找到导致治疗之间最大结果差异的因素。在这篇手稿中，我们展示了使用局部控制加 CART 的两步策略的使用，以确定相对于缓释文拉法辛，最有可能（最不可能）从度洛西汀治疗中受益的抑郁症患者。发现既往用药费用和年龄是与差异结果最相关的因素，在使用第二个数据集进行敏感性分析后，既往用药费用仍然是一个重要因素。版权所有 (c) 2013 John Wiley & Sons, Ltd.
Caregivers are regularly faced with decisions between competing treatments. Large observational health care databases provide a golden opportunity for research on heterogeneity in patient response to guide caregiver decisions, due to their sample size, diverse populations, and real-world setting. Local control is a promising tool for using observational data to detect patient subgroups with differential response on one treatment relative to another. While standard data mining approaches find subgroups with optimal responses for a particular population, detecting subgroups that reveal treatment differences while also adjusting for confounding in observational data is challenging. Local control utilizes unsupervised clustering to form non-parametric patient-level counterfactual treatment differences and displays them as an observed distribution of effect-size estimates. Classification and regression trees (CART) then find the factors that drive the greatest outcome differentiation between treatments. In this manuscript, we demonstrate the use of this two-step strategy using local control plus CART to identify depression patients most (least) likely to benefit from treatment with duloxetine relative to extended-release venlafaxine. Prior medication costs and age were found to be factors most associated with differential outcome, with prior medication costs remaining as an important factor after sensitivity analyses using a second dataset. Copyright (c) 2013 John Wiley & Sons, Ltd.