Real-World Efficacy and Safety Monitoring for Predicting Continuation of Tofacitinib Therapy in Patients with Ulcerative Colitis

Background Although the clinical efficacy of tofacitinib in patients with ulcerative colitis (UC) has been assessed in the OCTAVE trial, there is a lack of adequate data on its efficacy in real-world clinical settings. Aims To analyze the efficacy of tofacitinib and the predictors of its continuation. Methods Changes in clinical activity index (CAI), blood test results (C-reactive protein [CRP], albumin [Alb], and hemoglobin), and endoscopic scores (Mayo endoscopic subscore [MES], ulcerative colitis endoscopic index of severity [UCEIS]) were evaluated, and we investigated the factors that affect the rate and continuity of tofacitinib. Results Twenty-two patients with UC who were treated with tofacitinib were enrolled. Tofacitinib was continued in 16/22 (72.7%) patients. CAI significantly improved 4 weeks after tofacitinib induction (P < 0.01). In the blood tests, only Alb level improved significantly at week 2 compared with baseline (P = 0.03). In the non-failure group, serum Alb and CRP levels improved significantly from week 0 to week 24; however, similar changes were not observed in the failure group. After 6 months, the overall MES and UCEIS had significantly improved (P = 0.03 and P = 0.02, respectively). Kaplan-Meier analysis demonstrated that those with baseline UCEIS >= 5 had significantly lower tofacitinib continuation rate than those with baseline UCEIS <= 4, suggesting that baseline UCEIS may be a predictor of tofacitinib continuation (log-rank test: P < 0.01). Conclusions Tofacitinib is a promising therapeutic agent for the induction and maintenance therapy in UC. Baseline UCEIS may predict its therapeutic effects.