Tofacitinib for patients with anti-TNF refractory ulcerative proctitis: a multicenter cohort study from the GETAID.

BACKGROUND: While ulcerative proctitis (UP) can dramatically impair quality-of-life, treatments efficacy has been poorly investigated in UP as it was historically excluded from phase 2/3 randomized controlled trials in ulcerative colitis. AIM: To assess the effectiveness and safety of tofacitinib for the treatment of UP. METHODS: We conducted a retrospective multicenter study in seventeen GETAID centers including consecutive patients with UP treated with tofacitinib. The primary endpoint was steroid-free remission between week 8 and week 14, defined as a partial Mayo score of 2 (and no individual subscore above 1). Secondary outcomes included clinical response and steroid-free remission after induction and at one year. RESULTS: All the 35 enrolled patients previously received anti-TNF therapy and 88.6% were exposed to at least two lines of biologics. At baseline, the median partial Mayo score was 7 (IQR[5.5-7]). After induction (W8-W14), 42.9% and 60.0% of patients achieved steroid-free remission and clinical response, respectively. At one year, the steroid-free clinical remission and clinical response rates were 39.4% and 45.5%, respectively, while 51.2% (17/33) were still receiving tofacitinib treatment. Survival without tofacitinib withdrawal was estimated at 50.4% (95%CI[35.5-71.6]) at one year. Only a lower partial Mayo at baseline was independently associated with remission at induction (Odds ratio (OR) = 0.56 for an increase of 1, 95% confidence interval (95%CI) [0.33-0.95], p = 0.03). Five (14.3%) adverse events were reported with one leading to treatment withdrawal (septic shock secondary to cholecystitis). CONCLUSION: Tofacitinib may offer a therapeutic option for patients with refractory UP.