From the Department of Anesthesiology, Perioperative and Pain Medicine, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts.
Anesth Analg. 2018 Oct;127(4):1066-1073. doi: 10.1213/ANE.0000000000002787.
In clinical research, the gold standard level of evidence is the randomized controlled trial (RCT). The availability of nonrandomized retrospective data is growing; however, a primary concern of analyzing such data is comparability of the treatment groups with respect to confounding variables. Propensity score matching (PSM) aims to equate treatment groups with respect to measured baseline covariates to achieve a comparison with reduced selection bias. It is a valuable statistical methodology that mimics the RCT, and it may create an "apples to apples" comparison while reducing bias due to confounding. PSM can improve the quality of anesthesia research and broaden the range of research opportunities. PSM is not necessarily a magic bullet for poor-quality data, but rather may allow the researcher to achieve balanced treatment groups similar to a RCT when high-quality observational data are available. PSM may be more appealing than the common approach of including confounders in a regression model because it allows for a more intuitive analysis of a treatment effect between 2 comparable groups.We present 5 steps that anesthesiologists can use to successfully implement PSM in their research with an example from the 2015 Pediatric National Surgical Quality Improvement Program: a validated, annually updated surgery and anesthesia pediatric database. The first step of PSM is to identify its feasibility with regard to the data at hand and ensure availability of data on any potential confounders. The second step is to obtain the set of propensity scores from a logistic regression model with treatment group as the outcome and the balancing factors as predictors. The third step is to match patients in the 2 treatment groups with similar propensity scores, balancing all factors. The fourth step is to assess the success of the matching with balance diagnostics, graphically or analytically. The fifth step is to apply appropriate statistical methodology using the propensity-matched data to compare outcomes among treatment groups.PSM is becoming an increasingly more popular statistical methodology in medical research. It often allows for improved evaluation of a treatment effect that may otherwise be invalid due to a lack of balance between the 2 treatment groups with regard to confounding variables. PSM may increase the level of evidence of a study and in turn increases the strength and generalizability of its results. Our step-by-step approach provides a useful strategy for anesthesiologists to implement PSM in their future research.
在临床研究中,金标准水平的证据是随机对照试验(RCT)。非随机回顾性数据的可用性正在增加;然而,分析此类数据的主要关注点是治疗组相对于混杂变量的可比性。倾向评分匹配(PSM)旨在通过测量基线协变量来使治疗组均等化,以实现减少选择偏倚的比较。它是一种有价值的统计方法,模拟 RCT,可以创建“苹果对苹果”的比较,同时减少由于混杂引起的偏差。PSM 可以提高麻醉研究的质量,并拓宽研究机会的范围。PSM 不一定是劣质数据的“万能药”,而是当高质量的观察数据可用时,它可以使研究人员实现类似于 RCT 的平衡治疗组。PSM 可能比将混杂因素纳入回归模型的常见方法更具吸引力,因为它允许对两个可比组之间的治疗效果进行更直观的分析。我们提出了麻醉师可以在研究中成功实施 PSM 的 5 个步骤,并用 2015 年小儿国家手术质量改进计划的一个例子来说明:一个经过验证的、每年更新的手术和麻醉儿科数据库。PSM 的第一步是确定其在手边数据方面的可行性,并确保有任何潜在混杂因素的数据。第二步是从以治疗组为结果、平衡因素为预测因子的逻辑回归模型中获得一组倾向得分。第三步是将两个治疗组中的患者与相似的倾向得分相匹配,平衡所有因素。第四步是通过图形或分析平衡诊断来评估匹配的成功。第五步是使用倾向匹配数据应用适当的统计方法来比较治疗组之间的结果。PSM 正在成为医学研究中越来越流行的统计方法。它通常允许更好地评估治疗效果,否则由于两个治疗组在混杂变量方面缺乏平衡,治疗效果可能无效。PSM 可以提高研究的证据水平,从而提高其结果的强度和普遍性。我们的分步方法为麻醉师在未来的研究中实施 PSM 提供了一个有用的策略。