Jung Sin-Ho, Chi Sang Ah, Ahn Hyun Joo
Department of Biostatistics and Bioinformatics, Duke University, Durham, NC, USA.
Department of Health Sciences and Technology, SAIHST, Sungkyunkwan University, Seoul, South Korea.
Biom J. 2019 May;61(3):698-713. doi: 10.1002/bimj.201800049. Epub 2019 Jan 7.
In this paper, we investigate K-group comparisons on survival endpoints for observational studies. In clinical databases for observational studies, treatment for patients are chosen with probabilities varying depending on their baseline characteristics. This often results in noncomparable treatment groups because of imbalance in baseline characteristics of patients among treatment groups. In order to overcome this issue, we conduct propensity analysis and match the subjects with similar propensity scores across treatment groups or compare weighted group means (or weighted survival curves for censored outcome variables) using the inverse probability weighting (IPW). To this end, multinomial logistic regression has been a popular propensity analysis method to estimate the weights. We propose to use decision tree method as an alternative propensity analysis due to its simplicity and robustness. We also propose IPW rank statistics, called Dunnett-type test and ANOVA-type test, to compare 3 or more treatment groups on survival endpoints. Using simulations, we evaluate the finite sample performance of the weighted rank statistics combined with these propensity analysis methods. We demonstrate these methods with a real data example. The IPW method also allows us for unbiased estimation of population parameters of each treatment group. In this paper, we limit our discussions to survival outcomes, but all the methods can be easily modified for any type of outcomes, such as binary or continuous variables.
在本文中,我们研究观察性研究中生存终点的K组比较。在观察性研究的临床数据库中,根据患者的基线特征,以不同概率选择对患者的治疗方法。这通常会导致治疗组之间不可比,因为各治疗组患者的基线特征存在不平衡。为了克服这个问题,我们进行倾向分析,并在各治疗组之间匹配具有相似倾向得分的受试者,或者使用逆概率加权(IPW)比较加权组均值(或针对删失结局变量的加权生存曲线)。为此,多项逻辑回归一直是一种常用的倾向分析方法来估计权重。由于决策树方法的简单性和稳健性,我们建议将其作为一种替代的倾向分析方法。我们还提出了IPW秩统计量,称为Dunnett型检验和方差分析型检验,以比较3个或更多治疗组在生存终点上的差异。通过模拟,我们评估了结合这些倾向分析方法的加权秩统计量的有限样本性能。我们用一个实际数据例子展示了这些方法。IPW方法还使我们能够对每个治疗组的总体参数进行无偏估计。在本文中,我们将讨论限于生存结局,但所有方法都可以很容易地修改用于任何类型的结局,如二元或连续变量。