Tofacitinib in Pediatric Ulcerative Colitis: A Retrospective Multicenter Experience

Area of Science:

• Pediatric Gastroenterology
• Immunology
• Pharmacology

Background:

• Limited pediatric data exist for tofacitinib in moderate-to-severe ulcerative colitis (UC).
• Tofacitinib is approved for adult UC treatment.
• This study addresses the need for pediatric effectiveness and safety data.

Purpose of the Study:

• To evaluate the effectiveness and safety of tofacitinib in pediatric patients with ulcerative colitis (UC).
• To determine corticosteroid-free clinical remission rates at weeks 8 and 24.
• To assess colectomy rates and adverse events in this pediatric cohort.

Main Methods:

• Retrospective review of 101 pediatric UC patients from 16 international centers.
• Primary outcome: week 8 corticosteroid-free clinical remission (Pediatric Ulcerative Colitis Activity Index <10).
• Secondary outcomes: clinical response, week 24 remission, colectomy, and adverse events.

Main Results:

• 16% of patients achieved corticosteroid-free remission by week 8; 23% achieved it by week 24.
• 30% demonstrated clinical response at week 8.
• 25% underwent colectomy; adverse events were minor, including one herpes zoster case.

Conclusions:

• Tofacitinib demonstrated effectiveness in a highly refractory pediatric UC cohort, with at least 16% achieving remission by week 8.
• Adverse events were minor and consistent with adult data.
• This study represents the largest real-world pediatric cohort evaluating tofacitinib for UC.