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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
Summary
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.
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