Effectiveness of tofacitinib as rescue therapy in biological-naive patients with steroid-refractory acute severe...

Tofacitinib, a Janus kinase inhibitor approved for moderate-to-severe ulcerative colitis, has limited data in acute severe ulcerative colitis (ASUC), particularly in biologic-naive patients. This study evaluated its safety and efficacy as second-line therapy after intravenous steroid failure.

The present study retrospectively evaluated patients with biological-naive, steroid-refractory ASUC from 11 tertiary care centers in India who received tofacitinib from January 2022 to June 2024. Clinical response [≥3-point and ≥30% partial Mayo score (PMS) reduction with ≥1-point rectal bleeding decrease] was assessed on days 7 and 10; clinical and steroid-free remission (PMS 0-1, with or without steroids) were assessed at weeks 8 and 24.

The present study recruited 52 biologic-naive patients (61.5% male, median age: 35 years) with steroid-refractory ASUC who were initiated on tofacitinib. A response to tofacitinib was observed within 1 week of therapy in 73.1% (38/52) of patients, while 90.4% (47/52) had a response within 10 days of therapy. At 8 and 24 weeks, 71.1% (37/52) and 67.3% (35/52) were in clinical remission, and 65.4% (34/52) and 61.5% (32/52) were in steroid-free clinical remission, respectively. Adverse events were reported in 17.3% (9/52) of cases, of which four required temporary (tuberculosis: 2, herpes zoster: 2) and one permanent drug discontinuation (herpes zoster: 1).

Tofacitinib is a promising second-line therapy for ASUC patients after the failure of steroid therapy, with greater than 60% of patients having steroid-free clinical remission at the 24th week of therapy. Further randomized trials are needed to validate the study's findings.