Meta-analysis: safety and efficacy of ustekinumab in pediatric inflammatory bowel disease patients with anti-TNF failure.

Abstract

Background & aims: Evidence for ustekinumab (UST) in pediatric inflammatory bowel disease (IBD) is limited, particularly in patients who do not respond to anti-tumor necrosis factor (anti-TNF) therapy. We conducted a meta-analysis to evaluate the efficacy and safety of UST in pediatric IBD after anti-TNF failure.

Methods: PubMed, Cochrane Library, Embase, and Web of Science were searched from inception through August 2025. We included studies reporting outcomes of UST in pediatric IBD patients with prior anti-TNF failure. Pooled estimates were calculated using fixed- or random-effects models according to heterogeneity (I²).

Results: Fifteen studies including 653 pediatric patients were analyzed. Pooled clinical remission rates were 57% (95% CI, 28%-86%) at 8-16 weeks and 68% (95% CI, 58%-79%) at week 52. Steroid-free clinical remission was 44% (95% CI, 38%-49%) at 8-16 weeks and 58% (95% CI, 45%-70%) at week 52. The pooled endoscopic remission rate was 42% (95% CI, 35%-49%) and a biochemical remission rate of 63% (95% CI, 40%-87%). Adverse events occurred in 20% of patients (95% CI, 9%-30%), and serious adverse events occurred in 1% (95% CI, 0%-2%). At week 52, clinical remission rates were 69% in Crohn's disease (CD) and 65% in ulcerative colitis (UC).

Conclusion: In pediatric IBD patients with prior anti-TNF failure, UST was associated with favorable pooled remission rates and an acceptable safety profile, although substantial inter-study heterogeneity warrants cautious interpretation. These findings support UST as a feasible therapeutic option in this refractory pediatric setting; while highly generalizable to pediatric CD, the findings for the UC subgroup offer important preliminary evidence that warrants cautious interpretation due to limited precision.