Abstract Background Data are scarce on the deep remission of tofacitinib (TOFA) in moderate-to severe ulcerative colitis (UC), however, it can fundamentally change disease course and behaviour. Methods This was a prospective cohort study, assessing the short-term effectiveness of TOFA in UC patients. Baseline was the day of induction of TOFA. Clinical, biochemical, endoscopic and histological activities were assessed at baseline and at week 8 and 12, and we collected one-year treatment persistence. Primary outcome was week 12 histological remission (HR), defined as Nancy score<1 and endoscopic Mayo score (eMayo)≤1. Secondary outcomes were week 12 corticosteroid-free remission (CSFR), defined as clinical remission (CR; partial Mayo score [pMayo]<2 with a rectal bleeding subscore of 0) and C-reactive protein (CRP) ≤5 mg/L and endoscopic remission (eMayo score ≤1), furthermore, one-year treatment persistence were assessed. Data was handled by intention to treat analysis. We performed multivariable logistic, Cox-regression and linear regression models to control potential confounders and to reduce bias. The most reliable models were selected based on ROC analyzes with highest area under the curve (AUC). Results A total of 75 moderate-to severe UC patients (mean age 38.4 ± 12.1; male/female ratio 32/44) were involved in our prospective cohort study. In total, 7 patients (9.2%) achieved histological remission, while 20 patients (26.7%) achieved endoscopic remission. We found, that one-year treatment persistence was strongly influenced by week 12 endoscopic remission (β=3.79 [95% CI: 1.21 – 6.37]), but not by HR (β=1.58 [95% CI: -0.74 – 3.89]). In total, 34 patients (44.7%) were in CSFR at week 12, and higher baseline pMayo score decreased (OR=0.64 [95% CI: 0.43 – 0.94]), while albumin increased (OR=1.33 [95% CI: 1.01 – 1.76]) the chance of achieving the outcome (AUC = 0.93). Conclusion Based on our prospective cohort study, short-term HR is still a hard-to-reach endpoint in moderate-to-severe UC on TOFA. We found, that one-year treatment persistence is influenced by endoscopic mucosal healing, but not by HR, however, it should be enhanced, that as only a few patients achieved HR, the necessary statistical power was lacking.
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