Tofacitinib in the treatment of moderate to severe ulcerative colitis in Colombian pediatric patients: Real world experience

Introduction: There are no studies on efficacy of tofacitinib for moderate-severe ulcerative colitis (UC) in pediatric patients in Latin America. The aim of this study was to describe the efficacy and safety, in real world, treated with tofacitinib in our setting. Materials and methods: Case series of pediatric patients with UC who received treatment with tofacitinib in induction phase for 8 weeks and then maintenance therapy between November 2021 and February 2023. Results: Four female patients, median age 14.5 (SD 2.1; RIQ 12.5-16.5) years, all with prior biologic exposure, all 4 with prior use of anti-TNF, and 2/4 with prior use of anti-integrin. Clinical, biochemical and endoscopic remission was obtained in 3/4 at induction. Information was obtained from 3 patients in 6-month maintenance, 2/3 remained in clinical, biochemical and endoscopic remission and 1/3 has not achieved biochemical or endoscopic remission. Information was obtained from 1 patient in 12-month maintenance, achieving clinical and biochemical remission, however, endoscopic remission has not been achieved. One patient was initiated for severe acute UC with risk of colectomy, with significant improvement after 7 days, reaching therapeutic objectives at induction. No serious adverse events were reported in any of the cases. Conclusions: Efficacy and safety are demonstrated with tofacitinib in pediatric patients. With high percentage of response in induction treatment, sustained over time, and safe. In the context of severe acute hospitalized UC, it has a role as a potential rescue therapy due to its rapid action.