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

Denise D Young1, Jennifer E Murphy2, Mallory Chavannes3

  • 1Division of Pediatric Gastroenterology, Hepatology and Nutrition, UH Rainbow Babies and Children's Hospital, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA.

Journal of Pediatric Gastroenterology and Nutrition
|February 16, 2026
PubMed

Insights

Tofacitinib effectively treats pediatric inflammatory bowel disease (IBD), showing significant clinical response and remission rates. This Janus kinase (JAK) inhibitor demonstrated safety and efficacy in young patients with IBD.

Area of Science:

  • Gastroenterology
  • Immunology
  • Pediatric Medicine

Background:

  • Tofacitinib (a Janus kinase-1 inhibitor) is approved for adult ulcerative colitis (UC).
  • Limited data exists on its use in pediatric inflammatory bowel disease (IBD).

Purpose of the Study:

  • Evaluate tofacitinib's efficacy and safety in pediatric IBD patients.
  • Assess clinical response, corticosteroid-free remission, and biochemical response.
  • Determine the overall safety profile of tofacitinib in this population.

Main Methods:

  • Retrospective multicenter study of 37 pediatric IBD patients (2018-2023).
  • Data collected included demographics, disease characteristics, prior treatments, clinical/laboratory markers, and adverse events.
  • Follow-up ranged from 8-28 weeks.

Main Results:

  • 52.9% achieved clinical response by 8-12 weeks, increasing to 58.8% by 20-28 weeks.
  • 41.2% achieved corticosteroid-free remission by 8-12 weeks, rising to 47.1% by 20-28 weeks.
  • Improvements in albumin and C-reactive protein (CRP) were observed; infection was a common adverse effect.

Conclusions:

  • Tofacitinib demonstrates effectiveness in treating pediatric IBD patients.
  • This study represents one of the largest evaluations of tofacitinib in this demographic in the US.
  • Results support tofacitinib as a viable treatment option for pediatric IBD.
Abstract

Keywords:
JAK inhibitorsmall‐molecule agentsulcerative colitis

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