Real-world efficacy of ustekinumab and vedolizumab in pediatric and young adult patients with inflammatory bowel disease.

Objectives: Ustekinumab and vedolizumab are used off-label for pediatric inflammatory bowel disease (PIBD). Real-world data are needed to determine their positioning and safety. To assess the (1) 6-month and 1-year clinical outcome on ustekinumab and vedolizumab in PIBD based on Pediatric Ulcerative Colitis Activity Index, short Pediatric Crohn's Disease Activity Index, and calprotectin, and (2) secondary measures including safety and endoscopy.

Methods: Retrospective chart review of children and young adults with inflammatory bowel disease (IBD) treated at a pediatric academic hospital and two outpatient practices. Patients <21 years old with IBD were included if they received ustekinumab or vedolizumab between January 2015 and June 2022.

Results: One hundred eight patients were included. Patients with ulcerative colitis or IBD-unclassified had significantly higher 1-year steroid-free clinical remission with vedolizumab compared to ustekinumab (61% vs. 32%, p = 0.04) with a similar but nonsignificant trend at 6 months (59% vs. 38%, p = 0.12). Most patients with Crohn's disease receiving vedolizumab and ustekinumab achieved 6-month clinical remission (63% vs. 68%, p = 0.70) and 1-year steroid-free remission (64% vs. 67%, p > 0.90). Most patients receiving vedolizumab or ustekinumab achieved calprotectin <250 μg/g at 6 months (73% vs. 61%, p = 0.20) and 1 year (60% vs. 63%, p = 0.90), respectively. A greater proportion of biologic-naïve patients received vedolizumab, whereas most patients on ustekinumab were biologic-exposed. Adverse events occurred in 6.6% and 10% of patients on vedolizumab and ustekinumab, respectively.

Conclusions: Ustekinumab and vedolizumab are safe and effective in achieving clinical remission at 6 months and 1 year in PIBD. Comparative efficacy clinical trials are needed.