Supplementary Material for: Effectiveness and factors associated with response to golimumab in Japanese patients with ulcerative colitis in real clinical practice: Phoenix Study

Abstract Introduction: There have been limited reports on the clinical efficacy of golimumab (GLM) in Japanese patients with ulcerative colitis (UC) in real clinical practice. This study aimed to explore the real-life effectiveness and factors associated with response to GLM in Japanese patients with UC. Methods: This observational, retrospective, multicenter study was conducted in hospitals with expertise in inflammatory bowel disease treatment. Sixty-three patients treated with GLM and active UC were included in the analysis. Clinical remission (CR) (partial Mayo score ≤2) in the induction and maintenance phases after GLM treatment and associated factors were evaluated. Results: The proportion of patients achieving CR in the induction and maintenance phases was 41.3% (26/63) and 46.0% (29/63, the last observation carried forward method was used for patients who discontinued treatment for reasons other than inadequate response), respectively. The median partial Mayo score was 5 (IQR 4-6) at baseline, 3 (IQR 1-5) in the induction phase, and 1 (IQR 0-3) in the maintenance phase. Hemoglobin, platelet, and C-reactive protein levels changed, consistent with the partial Mayo score. Multivariate logistic analysis revealed that biologic-naive status was an independent factor associated with CR in the induction (p=0.0200) and maintenance (p=0.0459) phases, and a disease duration of >60 months until GLM initiation was associated with CR in the induction phase (p=0.0427). Conclusions: The effectiveness of GLM in daily clinical practice has been confirmed in Japanese patients with active UC. Biologic-naive patients responded more to GLM in the induction and maintenance phases, and patients with a disease duration of >60 months until initiation of GLM were more responsive in the induction phase.

引言：目前关于戈利木单抗（golimumab, GLM）在日本溃疡性结肠炎（ulcerative colitis, UC）患者真实临床实践中的临床疗效报道较为有限。本研究旨在探讨日本活动性溃疡性结肠炎患者使用戈利木单抗的真实世界疗效，以及与治疗应答相关的影响因素。方法：本研究为观察性回顾性多中心研究，在具备炎症性肠病治疗专长的医院开展。共纳入63例接受戈利木单抗治疗的活动性溃疡性结肠炎患者进行分析。评估戈利木单抗治疗后诱导治疗阶段与维持治疗阶段的临床缓解（clinical remission, CR，即部分Mayo评分≤2分）情况及其相关影响因素。结果：诱导治疗阶段与维持治疗阶段达到临床缓解的患者比例分别为41.3%（26/63）与46.0%（29/63，对于因应答不足以外的原因中断治疗的患者采用末次观测结转法进行数据填补）。基线时患者的中位部分Mayo评分为5（四分位数间距interquartile range, IQR 4~6），诱导治疗阶段为3（IQR 1~5），维持治疗阶段为1（IQR 0~3）。血红蛋白、血小板及C反应蛋白（C-reactive protein）水平的变化趋势与部分Mayo评分一致。多因素logistic回归分析（multivariate logistic analysis）显示，生物制剂初治状态是诱导治疗阶段（p=0.0200）与维持治疗阶段（p=0.0459）达到临床缓解的独立影响因素；而至开始使用戈利木单抗时病程超过60个月则与诱导治疗阶段的临床缓解相关（p=0.0427）。结论：本研究证实，戈利木单抗在日本活动性溃疡性结肠炎患者的日常临床实践中具有确切疗效。生物制剂初治患者在诱导治疗与维持治疗阶段对戈利木单抗的应答更佳，而至开始使用戈利木单抗时病程超过60个月的患者在诱导治疗阶段的应答更优。