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Robust estimation of heterogeneous treatment effects using electronic health record data
Statistics in Medicine ( IF 1.8 ) Pub Date : 2021-03-19 , DOI: 10.1002/sim.8926
Ruohong Li 1 , Honglang Wang 2 , Wanzhu Tu 1
Affiliation  

Estimation of heterogeneous treatment effects is an essential component of precision medicine. Model and algorithm‐based methods have been developed within the causal inference framework to achieve valid estimation and inference. Existing methods such as the A‐learner, R‐learner, modified covariates method (with and without efficiency augmentation), inverse propensity score weighting, and augmented inverse propensity score weighting have been proposed mostly under the square error loss function. The performance of these methods in the presence of data irregularity and high dimensionality, such as that encountered in electronic health record (EHR) data analysis, has been less studied. In this research, we describe a general formulation that unifies many of the existing learners through a common score function. The new formulation allows the incorporation of least absolute deviation (LAD) regression and dimension reduction techniques to counter the challenges in EHR data analysis. We show that under a set of mild regularity conditions, the resultant estimator has an asymptotic normal distribution. Within this framework, we proposed two specific estimators for EHR analysis based on weighted LAD with penalties for sparsity and smoothness simultaneously. Our simulation studies show that the proposed methods are more robust to outliers under various circumstances. We use these methods to assess the blood pressure‐lowering effects of two commonly used antihypertensive therapies.

中文翻译:

使用电子健康记录数据对异质治疗效果进行可靠的估计

评估异质性治疗效果是精密医学的重要组成部分。在因果推断框架内开发了基于模型和算法的方法,以实现有效的估计和推断。现有的方法,例如A学习器,R学习器,改进的协变量方法(带或不带效率增强),逆倾向得分加权和增强的逆倾向得分加权,大多是在平方误差损失函数下提出的。这些方法在存在数据不规则和高维度的情况下(例如在电子健康记录(EHR)数据分析中遇到的情况)的性能研究较少。在这项研究中,我们描述了一个通用的表述,该表述通过一个公共分数函数将许多现有的学习者统一起来。新的公式允许结合最小绝对偏差(LAD)回归和降维技术来应对EHR数据分析中的挑战。我们表明,在一组适度的规律性条件下,结果估计量具有渐近正态分布。在此框架内,我们提出了两种基于加权LAD的EHR分析的特定估计量,同时对稀疏性和平滑度进行了惩罚。我们的仿真研究表明,所提出的方法在各种情况下对于离群值均更健壮。我们使用这些方法来评估两种常用的降压疗法的降压效果。我们表明,在一组适度的规律性条件下,结果估计量具有渐近正态分布。在此框架内,我们提出了两种基于加权LAD的EHR分析的特定估计量,同时对稀疏性和平滑度进行了惩罚。我们的仿真研究表明,所提出的方法在各种情况下对于离群值均更健壮。我们使用这些方法来评估两种常用的降压疗法的降压效果。我们表明,在一组适度的规律性条件下,结果估计量具有渐近正态分布。在此框架内,我们提出了两种基于加权LAD的EHR分析的特定估计量,同时对稀疏性和平滑度进行了惩罚。我们的仿真研究表明,所提出的方法在各种情况下对于离群值均更健壮。我们使用这些方法来评估两种常用的降压疗法的降压效果。
更新日期:2021-05-08
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