Li Ruohong, Wang Honglang, Zhao Yi, Su Jing, Tu Wanzhu
Department of Biostatistics and Health Data Science, Indiana University School of Medicine.
Fairbanks School of Public Health.
Commun Stat Simul Comput. 2023;52(10):4981-4998. doi: 10.1080/03610918.2021.1974883. Epub 2021 Sep 14.
Heterogeneous treatment effect estimation is an essential element in the practice of tailoring treatment to suit the characteristics of individual patients. Most existing methods are not sufficiently robust against data irregularities. To enhance the robustness of the existing methods, we recently put forward a general estimating equation that unifies many existing learners. But the performance of model-based learners depends heavily on the correctness of the underlying treatment effect model. This paper addresses this vulnerability by converting the treatment effect estimation to a weighted supervised learning problem. We combine the general estimating equation with supervised learning algorithms, such as the gradient boosting machine, random forest, and artificial neural network, with appropriate modifications. This extension retains the estimators' robustness while enhancing their flexibility and scalability. Simulation shows that the algorithm-based estimation methods outperform their model-based counterparts in the presence of nonlinearity and non-additivity. We developed an package, , for public access to the proposed methods. To illustrate the methods, we present a real data example to compare the blood pressure-lowering effects of two classes of antihypertensive agents.
异质性治疗效果估计是根据个体患者特征量身定制治疗方案实践中的一个重要元素。大多数现有方法对数据不规则性的鲁棒性不足。为了增强现有方法的鲁棒性,我们最近提出了一个统一许多现有学习器的通用估计方程。但是基于模型的学习器的性能在很大程度上取决于潜在治疗效果模型的正确性。本文通过将治疗效果估计转化为加权监督学习问题来解决这一弱点。我们将通用估计方程与监督学习算法(如梯度提升机、随机森林和人工神经网络)相结合,并进行适当修改。这种扩展在增强估计器灵活性和可扩展性的同时,保留了其鲁棒性。模拟表明,在存在非线性和非可加性的情况下,基于算法的估计方法优于基于模型的方法。我们开发了一个名为 的软件包,供公众使用所提出的方法。为了说明这些方法,我们给出一个真实数据示例,比较两类抗高血压药物的降压效果。