ArticleAlimentary pharmacology & therapeutics2022
Children included in randomised controlled trials of biologics in inflammatory bowel diseases do not represent the real-world patient mix.
Article in Alimentary pharmacology & therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.
- Management of paediatric ulcerative colitis, part 1: Ambulatory care-An updated evidence-based consensus guideline from the European Society of Paediatric Gastroenterology, Hepatology and Nutrition and the European Crohn's and Colitis Organisation.Journal of pediatric gastroenterology and nutrition · 2025Guideline
- Effectiveness and safety of ustekinumab in pediatric Crohn's disease: Results of the REALITI study.Journal of pediatric gastroenterology and nutrition · 2026Observational
- Therapy de-escalation in paediatric patients with inflammatory bowel disease in remission: Data analysis from the CEDATA registry.Journal of pediatric gastroenterology and nutrition · 2026Article
- Association of Serum Levels of Ustekinumab, Vedolizumab, and Faecal Calprotectin in Paediatric Patients with Inflammatory Bowel Diseases: A Prospective Observational Study.Paediatric drugs · 2025Observational
- Clinical trials and young adults with inflammatory bowel disease.Health care transitions · 2025Article
- Tryptophan metabolites profile predict remission with dietary therapy in pediatric Crohn's disease.Therapeutic advances in gastroenterology · 2025Article
- Improved Clinical Outcomes With Early Anti-Tumour Necrosis Factor Alpha Therapy in Children With Newly Diagnosed Crohn's Disease: Real-world Data from the International Prospective PIBD-SETQuality Inception Cohort Study.Journal of Crohn's & colitis · 2024Observational
- A Real-World Prospective Cohort Study of Patients With Newly Diagnosed Crohn's Disease Treated by a Multidisciplinary Team: 1-Year Outcomes.Crohn's & colitis 360 · 2023Article
- 90K/Mac-2 BP Is a New Predictive Biomarker of Response to Infliximab Therapy in IBD Patients.International journal of molecular sciences · 2023Observational
- Children included in randomised controlled trials of biologics in inflammatory bowel diseases do not represent the real-world patient mix.Alimentary pharmacology & therapeutics · 2022Article
Corrections and comments
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Authors and funding
13 authors at 7 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatients enrolled in randomised controlled trials (RCTs) may differ from the target population due to restricted eligibility criteria.
aimTo compare treatment response to biologics in routine practice for children with inflammatory bowel diseases (IBD) who would and would not have been eligible for enrolment in the regulatory RCT of the same drug.
methodsWe enrolled children with IBD who initiated adalimumab, infliximab, vedolizumab or ustekinumab. The eligibility criteria as defined in the RCT of the corresponding biologic were applied to each patient. The primary outcome was 12-month steroid-free remission (SFR) without switching biologics or undergoing surgery.
resultsWe screened 289 children (198 [68%] with Crohn's disease [CD], 91 [32%] with ulcerative colitis [UC]) with 326 initiations of biologics. Only 62 of 164 (38%) children with moderate-to-severe disease would have been eligible for inclusion in the original RCTs. The SFR rate was higher in the eligible children (51%) than in the ineligible children (31%; OR 2.3 [95%CI 1.2-4.5]; p = 0.01). The main exclusion criterion was prohibited previous therapies (47%). Ineligible CD patients were older, more often had a family history of IBD and had higher levels of CRP than eligible children; in UC there were no differences between the groups.
conclusionMost children with IBD who initiate biologics would not have been eligible to be included in the corresponding regulatory RCTs. The outcomes of ineligible patients were worse than for eligible patients. Results from RCTs should be interpreted with caution when applied to clinical practice.
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