BACKGROUND: Tofacitinib has emerged as a new potential treatment for acute severe ulcerative colitis (ASUC). We conducted a systematic review to assess efficacy, safety, and integration in ASUC algorithms.

METHODS: Systematic search in MEDLINE, EMBASE, Cochrane Library, and Clinicaltrials.gov until August 17, 2022, including all studies reporting original observations on tofacitinib for ASUC, preferably defined according to Truelove and Witts criteria. Primary outcome was colectomy-free survival.

RESULTS: Of 1,072 publications identified, 21 studies were included of which three were ongoing clinical trials. The remaining comprised a pooled cohort originating from 15 case publications (n=42), a GETAID cohort study (n=55), a case-control study (n=40 cases), and a pediatric cohort (n=11). Of these 148 reported cases, tofacitinib was used as second line treatment after steroid failure in previous infliximab failures or third line after sequential steroid and infliximab or cyclosporine failure, 69 (47%) were female, age median ranged 17-34 years, disease duration 0.7-10 years. Overall, 30-day colectomy-free survival was 85% (n=123 of 145; n=3 without colectomy had follow-up <30 days), 90-day 86% (n=113 of 132; n=16 follow-up <90 days), and 180-day 69% (n=77 of 112; n=36 follow-up <180 days). Tofacitinib persistence at follow-up was 68-91%, clinical remission 35-69%, and endoscopic remission 55% reported. Adverse events occurred in 22 patients, predominantly being infectious complications other than herpes zoster (n=13), and resulted in tofacitinib discontinuation in seven patients.

CONCLUSION: Tofacitinib appears promising for treatment of ASUC with high short-term colectomy-free survival among refractory patients otherwise deemed for colectomy. However, large high-quality studies are needed.