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Japanese pediatric patient with refractory steroid-resistant ulcerative colitis successfully treated with
Toshihiko Kakiuchi1, Masato Yoshiura
1Department of Pediatrics, Faculty of Medicine, Saga University, Saga, Japan.
Insights
Tofacitinib successfully induced and maintained remission in a pediatric patient with moderately active ulcerative colitis (UC) refractory to other treatments. This case highlights tofacitinib as a safe and effective option for pediatric UC management.
Area of Science:
- Pediatric gastroenterology
- Immunomodulatory therapies
- Inflammatory bowel disease research
Background:
- Tofacitinib, a Janus kinase inhibitor, shows efficacy in adult ulcerative colitis (UC).
- Limited data exists on tofacitinib's efficacy in pediatric UC patients.
- Current treatment options for refractory pediatric UC are often limited.
Observation:
- A 9-year-old boy presented with a 2-month history of diarrhea and bloody stools.
- Colonoscopy confirmed total colitis-type UC with a pediatric UC activity index score of 40 (moderately active).
- The patient was refractory to multiple biologic therapies including vedolizumab, golimumab, and ustekinumab.
Findings:
- Tofacitinib 5 mg twice daily was administered for steroid-resistant UC.
- The patient achieved and maintained clinical remission of UC without adverse events.
- This represents the first reported pediatric case of tofacitinib-induced UC remission in Japan.
Implications:
- Tofacitinib demonstrates safety and efficacy in pediatric patients with moderately active UC.
- It offers a potential therapeutic option for steroid-dependent UC refractory to other biologics.
- Lower-dose tofacitinib induction therapy may be sufficient, potentially reducing adverse events in children and adults.
Rationale:
Tofacitinib is an orally administered selective Janus kinase inhibitor. Its efficacy and safety in adults with moderately to severely active ulcerative colitis (UC) have been evaluated in clinical trials; however, its efficacy in pediatric patients with UC is limited.
Patient Concerns:
A 9-years-old boy was referred to our hospital with chief complaints of diarrhea and bloody stool for 2 months. Colonoscopy revealed total colitis-type UC. His pediatric UC activity index score was 40, indicating moderately active UC.
Diagnosis:
UC.
Interventions:
Vedolizumab, golimumab, and ustekinumab were introduced because of steroid-resistant refractory UC; however, none of these biologics were effective or the effect was short-lived. Therefore, tofacitinib was administered 5 mg twice a day.
Outcomes:
The patient achieved UC remission after tofacitinib treatment, leading to maintained remission without adverse events.
Lessons:
To the best of our knowledge, this is the first pediatric case of moderately active UC that was successfully treated with tofacitinib in Japan. Tofacitinib is a safe drug for pediatric patients with moderately active UC. Even in steroid-dependent cases refractory to other biologics, tofacitinib can result in remission induction and maintenance effects. In children and adults, high-dose tofacitinib during induction therapy may be unnecessary to reduce adverse events.
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