Jingchao Sun, Scott Duncan, Subhadip Pal, Maiying Kong
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引用次数: 0
Abstract
Observational data, such as electronic clinical records and claims data, can prove invaluable for evaluating the Average Treatment Effect (ATE) and supporting decision-making, provided they are employed correctly. The Inverse Probability of Treatment Weighting (IPTW) method, based on propensity scores, has demonstrated remarkable efficacy in estimating ATE, assuming that the assumptions of exchangeability, consistency, and positivity are met. Directed Acyclic Graphs (DAGs) offer a practical approach to assess the exchangeability assumption, which asserts that treatment assignment and potential outcomes are independent given a set of confounding variables that block all backdoor paths from treatment assignment to potential outcomes. To ensure a consistent ATE estimator, one can adjust for a minimally sufficient adjustment set of confounding variables that block all backdoor paths from treatment assignment to the outcome. To enhance the efficiency of ATE estimators, our proposal involves incorporating both the minimally sufficient adjustment set of confounding variables and predictors into the propensity score model. Extensive simulations were conducted to evaluate the performance of propensity score-based IPTW methods in estimating ATE when different sets of covariates were included in the propensity score models. The simulation results underscored the significance of including the minimally sufficient adjustment set of confounding variables along with predictors in the propensity score models to obtain a consistent and efficient ATE estimator. We applied this proposed method to investigate whether tracheostomy was causally associated with in-hospital infant mortality, utilizing the 2016 Healthcare Cost and Utilization Project Kids' Inpatient Database. The estimated ATE was found to be approximately 2.30%-2.46% with p-value >0.05.
观察性数据,如电子临床记录和理赔数据,只要使用得当,在评估平均治疗效果(ATE)和支持决策方面可证明是无价之宝。基于倾向评分的反向治疗概率加权法(IPTW)已在估计 ATE 方面取得了显著成效,前提是满足可交换性、一致性和积极性等假设条件。有向无环图(DAG)为评估可交换性假设提供了一种实用方法,该假设认为,在一组混杂变量阻断了从治疗分配到潜在结果的所有后门路径的情况下,治疗分配和潜在结果是独立的。为确保 ATE 估计结果的一致性,我们可以对阻断从治疗分配到结果的所有后门路径的混杂变量进行最小充分的调整。为了提高 ATE 估计器的效率,我们的建议是将混杂变量和预测因子的最小充分调整集纳入倾向评分模型。我们进行了大量模拟,以评估当倾向得分模型中包含不同的协变量时,基于倾向得分的 IPTW 方法在估计 ATE 方面的表现。模拟结果表明,在倾向评分模型中加入最小充分的混杂变量调整集和预测因子对于获得一致且高效的 ATE 估计结果具有重要意义。我们利用 2016 年医疗成本与利用项目儿童住院病人数据库,将这一建议方法用于调查气管切开术是否与院内婴儿死亡率存在因果关系。结果发现,估计的 ATE 约为 2.30%-2.46%,P 值大于 0.05。
期刊介绍:
The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers:
Drug, device, and biological research and development;
Drug screening and drug design;
Assessment of pharmacological activity;
Pharmaceutical formulation and scale-up;
Preclinical safety assessment;
Bioavailability, bioequivalence, and pharmacokinetics;
Phase, I, II, and III clinical development including complex innovative designs;
Premarket approval assessment of clinical safety;
Postmarketing surveillance;
Big data and artificial intelligence and applications.