标题： 基于伪结果的框架分析电子健康记录中生存数据的治疗异质性 显示英文标题

标题： A pseudo-outcome-based framework to analyze treatment heterogeneity in survival data using electronic health records

摘要： 精准医疗的一个重要方面集中在由于患者特征的独特性而产生的治疗反应多样性上，这亦被称为异质性治疗效果（HTE），并识别出具有增强治疗效果的有益亚组。 在观察研究中使用右删失数据估计HTE仍然充满挑战。 本文提出了一种基于伪效应量的框架来分析生存数据中的HTE，该框架包括一系列用于估计HTE的元学习器，一个用于识别预测HTE变量的变量重要性度量，以及一个自适应的数据选择程序以选择具有增强治疗效果的亚组。 我们在多种观察研究设置下评估了该框架的有限样本性能。 此外，我们应用所提出的方法，利用一个大型哮喘电子健康记录数据集（该数据集包含了从新冠疫情前到后的就诊记录扩展）来分析书面哮喘行动计划（WAAP）对因哮喘加重导致的时间至急诊科（ED）回归的治疗异质性。 我们确定了哮喘结局较差但从WAAP中获益更多的脆弱患者亚组，并刻画了患者特征。 我们的研究为医疗保健提供者提供了宝贵的见解，特别是在公共健康危机期间如何战略性地分发WAAP，最终改善儿童哮喘的管理和控制。 显示英文摘要

摘要： An important aspect of precision medicine focuses on characterizing diverse responses to treatment due to unique patient characteristics, also known as heterogeneous treatment effects (HTE), and identifying beneficial subgroups with enhanced treatment effects. Estimating HTE with right-censored data in observational studies remains challenging. In this paper, we propose a pseudo-outcome-based framework for analyzing HTE in survival data, which includes a list of meta-learners for estimating HTE, a variable importance metric for identifying predictive variables to HTE, and a data-adaptive procedure to select subgroups with enhanced treatment effects. We evaluate the finite sample performance of the framework under various settings of observational studies. Furthermore, we applied the proposed methods to analyze the treatment heterogeneity of a Written Asthma Action Plan (WAAP) on time-to-ED (Emergency Department) return due to asthma exacerbation using a large asthma electronic health records dataset with visit records expanded from pre- to post-COVID-19 pandemic. We identified vulnerable subgroups of patients with poorer asthma outcomes but enhanced benefits from WAAP and characterized patient profiles. Our research provides valuable insights for healthcare providers on the strategic distribution of WAAP, particularly during disruptive public health crises, ultimately improving the management and control of pediatric asthma.