Effectiveness and Safety of Ustekinumab for Pediatric Inflammatory Bowel Disease: A Systematic Review
Shengbo Fang1, Sixi Zhang1, Chunyan Zhang2
1Department of Clinical Pharmacy, First Hospital of Jilin University, 71 Xinmin Street, Changchun, 130021, Jilin, China.
Insights
Ustekinumab shows potential for pediatric inflammatory bowel disease (IBD), with about half of patients achieving remission at one year. Further high-quality research is needed to confirm long-term benefits and optimize dosing for Crohn's disease and ulcerative colitis.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Ustekinumab use in pediatric inflammatory bowel disease (IBD) is off-label with limited existing data.
- This systematic review evaluates the efficacy and safety of ustekinumab in pediatric IBD patients.
Conclusions:
- Ustekinumab demonstrates a reasonable safety profile in pediatric IBD, with approximately half of patients achieving remission at one year.
- Dose optimization is frequently necessary, and low-quality evidence highlights the need for more high-quality data.
- Further research is required to establish long-term benefits and efficacy in pediatric CD and UC/IBD-U.
Background:
The use of ustekinumab in pediatric patients with inflammatory bowel disease (IBD) is off-label and the data are limited. We conducted a systematic review evaluating the efficacy and safety of ustekinumab in pediatric IBD.
Methods:
We systematically searched PubMed, EMBASE and Cochrane databases for studies of ustekinumab in children and adolescents with IBD investigating clinical remission, clinical response, corticosteroid-free (CS-free) remission, endoscopic remission/response, or safety up to March 17, 2023. A random-effects model was used for calculating summary estimates.
Results:
Eleven studies, comprising 370 patients were included. For Crohn's disease (CD), the pooled clinical remission rates were 34% (73/204) at 8-16 weeks and 46% (60/129) at 1 year. The pooled CS-free clinical remission rates were 23% (10/44) at 8-16 weeks and 45% (42/96) at 1 year. For ulcerative colitis (UC)/IBD unspecified (IBD-U), the pooled CS-free clinical remission rates were 24% (6/25) at 26 weeks and 46% (16/35) at 1 year. Endoscopic remission was found in 0-37.5% of CD and 63.6% of UC. Serious adverse events were reported in 3.5% of patients. About one half of patients required reduction in dose intervals and 62.75% patients could continue ustekinumab therapy at 1 year or final visit.
Conclusions:
According to low-quality evidence mainly from cohort studies and case series, approximately one half of patients with CD and UC/IBD-U achieved remission at 1 year. Ustekinumab has a reasonable safety profile and dose optimization is frequently required. Data on the long-term benefit and high-quality evidence are still needed.
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Inflammatory bowel disease, commonly known as IBD, refers to a collection of disorders that lead to persistent inflammation of the gastrointestinal tract. The two types of IBD are ulcerative colitis, which impacts the colon, and Crohn's disease, which can involve any part of the gastrointestinal segment.
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