Tofacitinib 20 vs 30 mg in acute severe ulcerative colitis: a double-blind randomized controlled trial.

BACKGROUND & AIMS: Tofacitinib has emerged as a promising therapy for acute severe ulcerative colitis (ASUC) due to its rapid onset of action. We compared the efficacy and safety of tofacitinib 20 mg/day vs 30 mg/day, both administered with intravenous corticosteroids (IVCS), in patients with ASUC.

METHODS

This was a double-blind, pilot, randomized controlled trial. Adult patients with ASUC were randomized to receive either tofacitinib 20 mg/day (Group 1) or 30 mg/day (Group 2) along with IVCS (hydrocortisone, 100 mg IV every 6 hours). The primary endpoint was clinical response by day 7. The secondary outcome was the cumulative probability of medical rescue therapy or post discharge escalation of therapy or colectomy within 90 days.

RESULTS

Seventy-two patients were randomized, with 36 allocated to each treatment group. At day 7, clinical response was achieved in 33/36 (91.7%) patients in Group 1 and 31/36 (86.1%) in Group 2(RR 1.06, 95% CI 0.90-1.25; P = .46). The absolute difference in response rates was 5.6% (95% CI -9.98% to 21.34%). Rescue therapy was required in 3 patients in Group 1 (all received infliximab) and 5 patients in Group 2 (2 underwent colectomy and 3 received infliximab). Event-free survival at 90 days, defined as the absence of rescue therapy, treatment escalation, or colectomy, was similar between groups (83.3% vs 80.2%; log-rank P = .74). Adverse events were predominantly mild, with no reported venous thromboembolism or major adverse cardiovascular events.

CONCLUSIONS

In this exploratory pilot trial, no statistically significant differences were observed with tofacitinib 30 mg/day compared with 20 mg/day when administered with IVCS.

CLINICAL TRIAL REGISTRATION NUMBER: Clinical Trials Registry-India: CTRI/2024/10/075098.