Supplementary Material for: Early phase partial Mayo score following golimumab treatment is associated with endoscopic improvement at 1-year in ulcerative colitis; A post hoc analysis of PURSUIT-J randomized controlled trial

Objective: Golimumab (GLM) is an anti-tumor necrosis factor-alpha antibody therapy for moderately to severely active ulcerative colitis (UC). Endoscopic improvement is considered one of UC treatment's main goals, and earlier prediction of future endosopic improvement has clinical implications. We retrospectively analyzed data from the PURSUIT-J, a phase III randomized controlled trial evaluating the efficacy of GLM in the maintenance phase, to find predictors for endoscopic improvement after 60 weeks of GLM treatment. Methods: Ninety-two patients who had completed the maintenance phase of the PURSUIT-J were divided into two groups: those with Mucosal healing (MHs : Mayo endoscopic subscore of 0 or 1) and those without MH at week 60 (non-MHs). Multivariate logistic regression analysis was conducted using baseline data in the induction phase to determine predictive factors for MHs compared to non-MHs. Results: Twenty-nine patients were classified as MHs and 63 as non-MHs. The multivariate logistic regression analysis showed that the odds ratio for partial Mayo (pMayo) score was highest in MHs (1.87 [95% CI 1.18–2.98]) at baseline in the induction phase. The receiver operating characteristic analysis to determine the timing of predictions of MHs using pMayo showed that an area under the curve reached 0.8 at week 14 after the first GLM administration. Conclusions: pMayo scores at week 14 of GLM treatment are associated with MH at week 60. These results suggest the timing of when a clinical decision to continue GLM based on the patient-reported outcomes and the physician’s general assessment could be considered.

研究目标：戈利木单抗（Golimumab，GLM）是一款抗肿瘤坏死因子-α（tumor necrosis factor-alpha, TNF-α）抗体疗法，用于治疗中重度活动性溃疡性结肠炎（ulcerative colitis, UC）。内镜改善被视为UC治疗的核心目标之一，提前预测未来内镜改善情况具有重要临床价值。本研究回顾性分析了PURSUIT-J试验的数据——该试验为评估GLM维持治疗疗效的III期随机对照试验，旨在探寻GLM治疗60周后内镜改善的预测因子。研究方法：将完成PURSUIT-J维持治疗的92例患者分为两组：第60周时达到黏膜愈合（mucosal healing, MH）的患者（记为MHs组，Mayo内镜亚评分为0或1）与未达到黏膜愈合的患者（记为non-MHs组）。采用诱导期的基线数据开展多因素logistic回归分析，以明确相较于non-MHs组，MHs组的预测因素。研究结果：最终有29例患者被归类为MHs组，63例为non-MHs组。多因素logistic回归分析显示，诱导期基线时的部分Mayo评分（partial Mayo score, pMayo）与MHs组的相关性最强，其比值比（odds ratio, OR）为1.87[95%置信区间（confidence interval, CI）：1.18~2.98]。通过pMayo评分预测MHs发生时机的受试者工作特征（Receiver Operating Characteristic, ROC）分析显示，在首次给予GLM后第14周，曲线下面积（area under the curve, AUC）达到0.8。研究结论：GLM治疗第14周时的pMayo评分与第60周时的黏膜愈合显著相关。上述结果提示，临床可考虑基于患者报告结局（patient-reported outcomes, PRO）及医师整体评估（physician's general assessment, PGA），在该时间点做出是否继续GLM治疗的决策。