Tofacitinib for ulcerative colitis in Brazil: a multicenter observational study on effectiveness and safety

Area of Science:

• Gastroenterology
• Immunology
• Pharmacology

Background:

• Ulcerative colitis (UC) is a chronic inflammatory bowel disease requiring effective long-term treatment options.
• Patients with refractory or difficult-to-treat UC often have limited therapeutic choices.
• Tofacitinib, a Janus kinase inhibitor, has emerged as a potential treatment for moderate to severe UC.

Purpose of the Study:

• To evaluate the real-world, long-term effectiveness and safety of tofacitinib in a large cohort of patients with refractory UC.
• To assess clinical and endoscopic outcomes, as well as colectomy rates, over a 30-month period.
• To identify factors associated with treatment outcomes, including colectomy risk.

Main Methods:

• A multicenter, retrospective, observational cohort study design was employed.
• Included patients with moderately to severely active UC who received tofacitinib for at least 8 weeks.
• Evaluated clinical remission, endoscopic response/remission, biochemical remission, steroid-free remission, loss of response, drug discontinuation, colectomy, and adverse events up to 30 months.

Main Results:

• 127 patients with UC were included, with 79.5% having previously failed biological therapy.
• Clinical remission rates at 12-16 weeks, 26 weeks, and 1 year were 31.5%, 46.5%, and 37.0%, respectively.
• Colectomy was required in 7.9% of patients, with a significantly higher rate in those with baseline serum albumin ≤3.5 g/dL (21.4% vs. 4.1%).

Conclusions:

• Tofacitinib demonstrated effectiveness in inducing clinical remission and endoscopic improvement in biologically refractory UC patients over 30 months.
• The drug showed a favorable safety profile and helped prevent colectomy in this real-world setting.
• Baseline hypoalbuminemia is a significant predictor of higher colectomy rates in UC patients treated with tofacitinib.