Real-world clinical and endoscopic outcomes after one year tofacitinib treatment in ulcerative colitis

Objective Tofacitinib, an oral Janus kinase inhibitor, is approved for the treatment of moderate to severe ulcerative colitis (UC). We evaluated clinical and endoscopic efficacy, safety and drug survival of tofacitinib up to one year in a real-world cohort. Methods In this retrospective cohort study, 36 UC patients were included who received tofacitinib. The primary outcome was combined with steroid-free clinical remission [Simple Clinical Colitis Activity Index (SCCAI) <= 2] and endoscopic improvement (Mayo score <= 1) at 52 weeks. Secondary outcomes included clinical (SCCAI drop >= 3) and endoscopic response (Mayo score drop >= 1), biochemical remission [fecal calprotectin (FC) <= 150 mg/kg and C-reactive protein <= 5 mg/L), safety and drug survival. Results Median disease duration was 7 (3-14) years and 89 and 42% of patients failed prior anti-tumor necrosis factor (anti-TNF) and vedolizumab treatment, respectively. Combined corticosteroid-free clinical remission and endoscopic improvement were observed in 8/36 patients (22%), 6/35 (17%) and 12/31 (39%), at 16, 36 and 52 weeks, respectively. Corresponding combined clinical and endoscopic response rates were 15/36 (42%), 12/35 (34%), 15/31 (48%) and biochemical remission rates were 11/33 (33%), 10/32 (31%) and 10/29 (34%). Nine infections (two herpes zoster) led to dose reduction or (temporary) drug withdrawal. Permanent withdrawal occurred in 14/36 patients (33%) after a median duration of 9 (5-30) weeks. Drug survival at 1 year was 60%. Patients that failed anti-TNF were less likely to discontinue tofacitinib treatment early compared to patients without prior anti-TNF use (hazard ratio 0.20, 95% confidence interval 0.06-0.65). Conclusion In a refractory UC population, combined steroid-free clinical remission and endoscopic improvement were found in 39% of patients at 1 year. Copyright (C) 2021 Wolters Kluwer Health, Inc. All rights reserved.