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Effectiveness and Safety of Ustekinumab for Pediatric Inflammatory Bowel Disease: A Systematic Review

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Abstract

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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Acknowledgments

Thanks to Professor Ying kit Leung who provided editorial assistance.

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Authors and Affiliations

  1. Department of Clinical Pharmacy, First Hospital of Jilin University, 71 Xinmin Street, Changchun, 130021, Jilin, China

    Shengbo Fang & Sixi Zhang

  2. Department of Pediatric Gastroenterology Unit, First Hospital of Jilin University, Changchun, China

    Chunyan Zhang & Libo Wang

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Correspondence to Libo Wang.

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The authors declare that they have no competing interests.

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All data used during the study are available from the corresponding author by request.

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Author contributions

SF and SZ performed screening of papers, data extraction, quality assessment, collected and analyzed the data. SF and CZ wrote and revised the manuscript. SF performed the literature search. LW developed the study concept and protocol, held the leading role in coordinating the project, senior authorship to resolve disagreements on inclusion, exclusion and quality assessment of papers, and critiqued and edited the manuscript. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.

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Fang, S., Zhang, S., Zhang, C. et al. Effectiveness and Safety of Ustekinumab for Pediatric Inflammatory Bowel Disease: A Systematic Review. Pediatr Drugs 25, 499–513 (2023). https://doi.org/10.1007/s40272-023-00586-7

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