ICES, Toronto, Ontario, Canada.
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.
Stat Med. 2021 Nov 10;40(25):5565-5586. doi: 10.1002/sim.9141. Epub 2021 Aug 10.
We describe a new method to combine propensity-score matching with regression adjustment in treatment-control studies when outcomes are binary by multiply imputing potential outcomes under control for the matched treated subjects. This enables the estimation of clinically meaningful measures of effect such as the risk difference. We used Monte Carlo simulation to explore the effect of the number of imputed potential outcomes under control for the matched treated subjects on inferences about the risk difference. We found that imputing potential outcomes under control (either single imputation or multiple imputation) resulted in a substantial reduction in bias compared with what was achieved using conventional nearest neighbor matching alone. Increasing the number of imputed potential outcomes under control resulted in more efficient estimation, with more efficient estimation of the estimated risk difference when increasing the number of the imputed potential outcomes. The greatest relative increase in efficiency was achieved by imputing five potential outcomes; once 20 outcomes under control were imputed for each matched treated subject, further improvements in efficiency were negligible. We also examined the effect of the number of these imputed potential outcomes on: (i) estimated standard errors; (ii) mean squared error; (iii) coverage of estimated confidence intervals. We illustrate the application of the method by estimating the effect on the risk of death within 1 year of prescribing beta-blockers to patients discharged from hospital with a diagnosis of heart failure.
我们描述了一种新方法,可在治疗-对照研究中将倾向评分匹配与回归调整相结合,当结果为二分类时,通过对匹配的治疗组个体的对照进行潜在结果的多次插补来实现。这使我们能够估计具有临床意义的效应量,如风险差。我们使用蒙特卡罗模拟来探讨匹配的治疗组个体的对照下潜在结果的插补数量对风险差推断的影响。我们发现,与仅使用传统最近邻匹配相比,对对照下的潜在结果进行插补(无论是单插补还是多插补)都会大大减少偏差。随着对照下潜在结果数量的增加,估计会更加有效,并且随着插补潜在结果数量的增加,对估计的风险差的估计也会更加有效。通过插补五个潜在结果可以实现最大的相对效率提高;一旦为每个匹配的治疗个体插补了 20 个对照下的结果,进一步提高效率的效果则微不足道。我们还研究了这些插补潜在结果的数量对以下方面的影响:(i)估计的标准误差;(ii)均方误差;(iii)估计置信区间的覆盖率。我们通过估计心力衰竭出院患者开处方β受体阻滞剂后 1 年内死亡风险的影响来说明该方法的应用。