Glynn Robert J, Schneeweiss Sebastian, Stürmer Til
Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Basic Clin Pharmacol Toxicol. 2006 Mar;98(3):253-9. doi: 10.1111/j.1742-7843.2006.pto_293.x.
Use of propensity scores to identify and control for confounding in observational studies that relate medications to outcomes has increased substantially in recent years. However, it remains unclear whether, and if so when, use of propensity scores provides estimates of drug effects that are less biased than those obtained from conventional multivariate models. In the great majority of published studies that have used both approaches, estimated effects from propensity score and regression methods have been similar. Simulation studies further suggest comparable performance of the two approaches in many settings. We discuss five reasons that favour use of propensity scores: the value of focus on indications for drug use; optimal matching strategies from alternative designs; improved control of confounding with scarce outcomes; ability to identify interactions between propensity of treatment and drug effects on outcomes; and correction for unobserved confounders via propensity score calibration. We describe alternative approaches to estimate and implement propensity scores and the limitations of the C-statistic for evaluation. Use of propensity scores will not correct biases from unmeasured confounders, but can aid in understanding determinants of drug use and lead to improved estimates of drug effects in some settings.
近年来,在将药物与结局相关联的观察性研究中,使用倾向得分来识别和控制混杂因素的情况大幅增加。然而,使用倾向得分是否以及在何时能提供比传统多变量模型所得出的药物效应估计值偏差更小的结果,仍不明确。在绝大多数同时使用这两种方法的已发表研究中,倾向得分法和回归法得出的估计效应相似。模拟研究进一步表明,在许多情况下这两种方法的表现相当。我们讨论了支持使用倾向得分的五个理由:关注药物使用指征的价值;来自替代设计的最优匹配策略;对稀缺结局混杂因素的更好控制;识别治疗倾向与药物对结局效应之间相互作用的能力;以及通过倾向得分校准对未观察到的混杂因素进行校正。我们描述了估计和实施倾向得分的替代方法以及用于评估的C统计量的局限性。使用倾向得分不会纠正未测量混杂因素造成的偏差,但有助于理解药物使用的决定因素,并在某些情况下改进药物效应的估计。