Gwon Yeongjin, Mo May, Chen Ming-Hui, Chi Zhiyi, Li Juan, Xia Amy H, Ibrahim Joseph G
Department of Biostatistics, University of Nebraska Medical Center, Omaha, Nebraska, USA.
Amgen Inc., Thousand Oaks, California, USA.
Stat Med. 2020 Mar 12. doi: 10.1002/sim.8518.
Crohn's disease (CD) is a life-long condition associated with recurrent relapses characterized by abdominal pain, weight loss, anemia, and persistent diarrhea. In the US, there are approximately 780 000 CD patients and 33 000 new cases added each year. In this article, we propose a new network meta-regression approach for modeling ordinal outcomes in order to assess the efficacy of treatments for CD. Specifically, we develop regression models based on aggregate covariates for the underlying cut points of the ordinal outcomes as well as for the variances of the random effects to capture heterogeneity across trials. Our proposed models are particularly useful for indirect comparisons of multiple treatments that have not been compared head-to-head within the network meta-analysis framework. Moreover, we introduce Pearson residuals and construct an invariant test statistic to evaluate goodness-of-fit in the setting of ordinal outcome data. A detailed case study demonstrating the usefulness of the proposed methodology is carried out using aggregate ordinal outcome data from 16 clinical trials for treating CD.
克罗恩病(CD)是一种终身性疾病,伴有反复发作,其特征为腹痛、体重减轻、贫血和持续性腹泻。在美国,约有78万克罗恩病患者,且每年新增3.3万病例。在本文中,我们提出一种用于对有序结果进行建模的新网络meta回归方法,以评估克罗恩病治疗方法的疗效。具体而言,我们基于聚合协变量开发回归模型,用于有序结果的潜在切点以及随机效应的方差,以捕捉各试验间的异质性。我们提出的模型对于在网络meta分析框架内未进行直接比较的多种治疗方法的间接比较特别有用。此外,我们引入Pearson残差并构建一个不变检验统计量,以评估有序结果数据背景下的拟合优度。使用来自16项治疗克罗恩病的临床试验的聚合有序结果数据进行了详细的案例研究,以证明所提出方法的实用性。