Multicenter experience using tofacitinib for treatment of refractory pediatric inflammatory bowel diseases in the United States

OBJECTIVES: Tofacitinib is a selective Janus kinase (JAK-1) inhibitor approved for the treatment of ulcerative colitis (UC) in adults. Data is limited in the pediatric population. The aim of this study was to evaluate the efficacy of tofacitinib in pediatric inflammatory bowel disease (IBD). The primary aim was to assess clinical response (categorical improvement in disease activity from baseline) after induction (8-12 weeks). The secondary aims were to assess effectiveness of tofacitinib in achieving corticosteroid-free remission and biochemical response and overall safety. METHODS: We performed a retrospective multicenter study of pediatric patients with IBD at eight centers across the United States between 2018 and 2023. We collected demographic data, disease classification, prior biologic medication use, laboratory and clinical disease activity, corticosteroid use, adverse events and side effects, and surgical data. RESULTS: Thirty-seven patients were included in this study. Eighteen patients (52.9%) achieved a clinical response within 8-12 weeks, which increased to 20 (58.8%) by 20-28 weeks. Corticosteroid-free remission was achieved by 14 patients (41.2%) by 8-12 weeks, followed by 16 patients (47.1%) at 20-28 weeks. Improvement in albumin and C-reactive protein (CRP) occurred as early as 8-12 weeks. Surgical rate was 10.5% (4/38 patients). Infection was a common adverse effect reported with the use of tofacitinib. CONCLUSION: This is one of the largest studies evaluating the use of tofacitinib in pediatric IBD in the United States. Our results demonstrate that tofacitinib is an effective treatment for these patients.