BACKGROUND Tofacitinib is a Janus kinase inhibitor approved for the treatment of ulcerative colitis (UC). AIM To evaluate effectiveness, safety and use of tofacitinib in daily practice. METHODS UC patients initiating tofacitinib were prospectively enrolled in 15 hospitals in the Netherlands. Corticosteroid-free clinical remission (short clinical colitis activity index [SCCAI] ≤2), biochemical remission (faecal calprotectin level ≤250 µg/g), combined corticosteroid-free clinical and biochemical remission, predictors of remission, safety outcomes, treatment dose and effect on lipids were determined at weeks 12 and 24. Endoscopic outcomes were evaluated in centres with routine endoscopic evaluation. RESULTS In total, 123 UC patients (95% anti-TNF, 62% vedolizumab and 3% ustekinumab experienced) were followed for a median duration of 24 weeks (interquartile range 12-26). The proportion of patients in corticosteroid-free clinical, biochemical, and combined corticosteroid-free clinical and biochemical remission rate at week 24 was 29% (n: 22/77), 25% (n: 14/57), and 19% (n: 11/57) respectively. Endoscopic remission (Mayo = 0) was achieved in 21% of patients at week 12 (n: 7/33). Prior vedolizumab exposure was associated with reduced clinical remission (odds ratio 0.33, 95% confidence interval [CI] 0.11-0.94). At week 24, 33% (n: 14/42) of patients still on tofacitinib treatment used 10 mg twice daily. In total, 33 tofacitinib-related adverse events (89 per 100 patient years) occurred, 7 (6% of total cohort) resulted in discontinuation. Cholesterol, HDL and LDL levels increased during induction treatment by 18% (95% CI 9-26), 18% (95% CI 8-28) and 21% (95% CI 14-39) respectively. CONCLUSION Tofacitinib is an effective treatment for UC after anti-TNF and vedolizumab failure. However, a relatively high rate of adverse events was observed resulting in discontinuation in 6% of patients.

中文翻译：

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溃疡性结肠炎的托法替尼：前瞻性荷兰克罗恩和结肠炎倡议 (ICC) 注册的结果。

背景托法替尼是一种被批准用于治疗溃疡性结肠炎 (UC) 的 Janus 激酶抑制剂。目的 评估托法替尼在日常实践中的有效性、安全性和使用。方法 荷兰 15 家医院前瞻性招募了开始使用托法替尼的 UC 患者。无皮质类固醇临床缓解（短期临床结肠炎活动指数 [SCCAI] ≤2）、生化缓解（粪便钙卫蛋白水平≤250 µg/g）、联合无皮质类固醇临床和生化缓解、缓解预测因子、安全性结果、治疗剂量和在第 12 周和第 24 周确定对脂质的影响。在常规内窥镜评估中心评估内窥镜结果。结果 总共有 123 名 UC 患者（95% 抗 TNF、62% 的 vedolizumab 和 3% 的 ustekinumab 经历了中位持续时间 24 周（四分位距 12-26）。第 24 周无皮质类固醇临床、生化和联合无皮质类固醇临床和生化缓解率的患者比例为 29% (n: 22/77)、25% (n: 14/57) 和 19% ( n: 11/57) 分别。21% 的患者在第 12 周（n：7/33）获得内窥镜缓解（Mayo = 0）。既往维多珠单抗暴露与临床缓解降低相关（优势比 0.33，95% 置信区间 [CI] 0.11-0.94）。在第 24 周时，仍有 33% (n: 14/42) 的患者仍在接受托法替尼治疗，每天两次使用 10 mg。总共发生了 33 起托法替尼相关的不良事件（每 100 患者年 89 起），7 起（占总队列的 6%）导致停药。胆固醇，HDL 和 LDL 水平在诱导治疗期间分别增加了 18% (95% CI 9-26)、18% (95% CI 8-28) 和 21% (95% CI 14-39)。结论 Tofacitinib 是治疗抗 TNF 和维多珠单抗失败后 UC 的有效方法。然而，观察到的不良事件发生率相对较高，导致 6% 的患者停药。