J Crohns Coliti. 2024 Aug 14;18(8):1250-1260. doi: 10.1093/ecco-jcc/jjae030.

Joel R Rosh ¹, Dan Turner ², Jeffrey S Hyams ³, Marla Dubinsky ⁴, Anne M Griffiths ⁵, Stanley A Cohen ⁶, Kim Hung Lo ⁷, Lilianne Kim ⁷, Sheri Volger ⁸, Renping Zhang ⁹, Richard Strauss ⁸, Laurie S Conklin ¹⁰

Author information

¹Division of Pediatric Gastroenterology, Cohen Children's Medical Center, New Hyde Park, NY, USA.

²Juliet Keidan Institute of Pediatric Gastroenterology, Shaare Zedek Medical Center, Hebrew University of Jerusalem, Israel.

³Division of Pediatric Gastroenterology, Connecticut Children's, Hartford, CT, USA.

⁴Division of Pediatric Gastroenterology, Mount Sinai Medical Center, New York, NY, USA.

⁵Division of Pediatric Gastroenterology, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.

⁶Division of Pediatric Gastroenterology, Children's Center for Digestive Health Care, Atlanta, GA, USA.

⁷Statistics and Decision Sciences, Janssen Research & Development, LLC, Spring House, PA, USA.

⁸Pediatric Development Team, Janssen Research & Development, Spring House, PA, USA.

⁹Data Analytics, Janssen Research & Development, Spring House, PA, USA.

¹⁰Child Health Innovation Leadership Department, Johnson & Johnson, New Brunswick, NJ, USA.

Abstract

Background and aims: Most paediatric inflammatory bowel disease [IBD] studies are performed after medications are approved in adults, and the majority of participants in these studies are adolescents. We hypothesised that adolescent-onset IBD is not fundamentally different from adult-onset IBD. If this is correct, the value of delaying access to novel drugs in adolescents becomes questioned.

Methods: Data from 11 randomised, double-blind, placebo-controlled, adult Phases 2 and 3 trials of four biologics were analysed. Participants were categorised as having adolescent- or adult-onset disease [diagnosed 12 to <18, or ≥18 years]. Multivariable modelling explored the association between age at diagnosis and response to treatment, after adjustment for disease duration, extent, and severity at baseline. Data from dose arms were pooled to evaluate similarity of therapeutic response between adolescent- and adult-onset IBD within the same trial [not between doses or across trials]. Ratios of odds ratios [ORs] between the two groups were evaluated.

Results: Data from 6283 study participants (2575 with Crohn's disease [CD], 3708 with ulcerative colitis [UC]) were evaluated. Of 2575 study participants with CD, 325 were 12-<18 years old at diagnosis; 836 participants [32.4%] received placebo. Of 3708 participants with UC, 221 were 12-<18 years old at diagnosis; 1212 [33%] were receiving placebo. The majority of the ratios of ORs were within 2-fold, suggesting that responses in adolescent- and adult-onset participants are generally similar.

Conclusion: Data presented lend support for extrapolating efficacy of biologics from adults to adolescents with IBD, which would facilitate earlier labelling and patient access.