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Upadacitinib for difficult-to-treat paediatric Crohn's disease

N Nedelkopoulou1, S Sharma1, M Khalid1

  • 1Sheffield Children's Hospital, NHS Foundation Trust Sheffield UK.

JPGN Reports
|August 15, 2025
PubMed

Insights

Upadacitinib shows promise for treating refractory paediatric Crohn's disease. This study highlights its effectiveness in two adolescents, suggesting a potential new treatment option for severe pediatric inflammatory bowel disease.

Area of Science:

  • Pediatric Gastroenterology
  • Inflammatory Bowel Disease Research
  • Pharmacological Treatments

Background:

  • Pediatric-onset inflammatory bowel disease (IBD) presents significant long-term healthcare challenges.
  • Existing treatments, including anti-tumour necrosis factor-alpha agents, necessitate expanded therapeutic options for young patients.
  • Limited pediatric data exist for upadacitinib, despite promising adult evidence.

Purpose of the Study:

  • To evaluate the efficacy and safety of upadacitinib in adolescents with highly refractory Crohn's disease.
  • To address the scarcity of pediatric data for this emerging therapeutic agent.

Main Methods:

  • Case report of two adolescent patients with severe Crohn's disease.
  • Treatment with upadacitinib in patients refractory to other therapies.
  • Inclusion of a patient with prior subtotal colectomy.

Main Results:

  • Upadacitinib demonstrated effective treatment in both adolescent patients.
  • Positive outcomes were observed even in a patient with a history of subtotal colectomy.

Conclusions:

  • Upadacitinib is a potentially effective treatment for highly refractory pediatric Crohn's disease.
  • This case series supports further investigation of upadacitinib in pediatric IBD populations.

Keywords:
JAK inhibitorsJanus kinase inhibitorsinflammatory bowel disease

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