Pilot trial finds no significant response difference between tofacitinib doses in severe ulcerative colitis

A double-blind pilot randomized controlled trial published in the Journal of Crohn's and Colitis compared two tofacitinib doses in 72 adults with acute severe ulcerative colitis. Participants received either 20 or 30 mg/day, with 36 patients per group, alongside intravenous hydrocortisone 100 mg every six hours. The primary endpoint was clinical response by day 7.

Clinical response occurred in 33/36 patients receiving 20 mg/day (91.7%) and 31/36 receiving 30 mg/day (86.1%; relative risk 1.06, 95% CI 0.90–1.25; P=.46). The absolute response difference was 5.6 percentage points (95% CI −9.98 to 21.34). Rescue therapy was required in three patients in the lower-dose group, all receiving infliximab, and five in the higher-dose group, including two who underwent colectomy and three receiving infliximab. Ninety-day event-free survival—absence of rescue therapy, treatment escalation, or colectomy—was 83.3% versus 80.2% (log-rank P=.74).

Adverse events were predominantly mild, with no reported venous thromboembolism or major adverse cardiovascular events. These exploratory findings do not demonstrate an advantage for 30 mg/day, but the small sample and wide confidence interval preclude establishing equivalence or excluding uncommon harms. Both groups received corticosteroids, so the trial does not determine tofacitinib's added benefit over corticosteroids alone. Only the abstract was available for this update.