Sarlon E, Millier A, Aballéa S, Toumi M
INSERM, U669, National Institute of Health and Medical Research, Paris, France,
Community Ment Health J. 2014 Aug;50(6):711-20. doi: 10.1007/s10597-014-9723-x. Epub 2014 Apr 3.
Although randomised controlled trials are regarded as the gold standard for treatments efficacy, evidence from observational studies remains relevant. To address the problem of possible confounding in these studies, investigators must employ analysis methods that adjust for confounders and lead to an unbiased estimation of the treatment effect. In this paper, the authors describe two relevant statistical methods. The first method represents the classical approach consisting of a multiple regression model including the effects of treatment and covariates. This approach considers the relation between prognostic factors and the outcome variable as a relevant criterion for adjustment. The second method is based on the propensity score, and focuses on the relation between prognostic factors and treatment assignment. These approaches were applied to a cohort of 183 French schizophrenic patients who were followed for a 2-year period (from 1998 to 2000). The probability of relapse according to antipsychotic treatment exposure was modelled using Cox regression models with the two statistical methods. Goodness-of-fit criteria were used to compare the modelling approaches. This study demonstrates that the propensity score, a predicted probability, has an important balancing property that underscores its value in strengthening the results of nonrandomised observational studies.
尽管随机对照试验被视为治疗效果的金标准,但观察性研究的证据仍然具有相关性。为了解决这些研究中可能存在的混杂问题,研究人员必须采用能够对混杂因素进行调整并对治疗效果进行无偏估计的分析方法。在本文中,作者描述了两种相关的统计方法。第一种方法是经典方法,由一个包含治疗效果和协变量的多元回归模型组成。这种方法将预后因素与结局变量之间的关系视为调整的相关标准。第二种方法基于倾向得分,侧重于预后因素与治疗分配之间的关系。这些方法应用于一组183名法国精神分裂症患者,随访期为2年(从1998年到2000年)。使用这两种统计方法,通过Cox回归模型对根据抗精神病药物治疗暴露情况的复发概率进行建模。采用拟合优度标准来比较建模方法。这项研究表明,倾向得分,即一个预测概率,具有重要的平衡特性,突出了其在加强非随机观察性研究结果方面的价值。