Effectiveness and Safety of Adalimumab in Patients With Very Early-Onset Inflammatory Bowel Disease: A Retrospective Study on Behalf of the Porto Inflammatory Bowel Disease Working Group of European Society for Pediatric Gastroenterology Hepatology and Nutrition
Status Publisher Language English Country Great Britain, England Media print-electronic
Document type Journal Article
PubMed
39813158
DOI
10.1093/ibd/izae302
PII: 7954627
Knihovny.cz E-resources
- Keywords
- VEO-IBD, adalimumab, anti-TNF, monogenic IBD,
- Publication type
- Journal Article MeSH
BACKGROUND AND AIMS: Patients with very early-onset inflammatory bowel disease (VEO-IBD), with an age of onset < 6 years, can present with severe manifestations and may require biologic therapy. Infliximab and adalimumab are approved for induction and maintenance in pediatric IBD patients but are licensed only above the age of 6 years. Effectiveness and safety data on adalimumab in this patient population are lacking. We assessed the therapeutic response to help close this gap. METHODS: This retrospective study involved 30 sites worldwide. Demographic, clinical, and laboratory data were collected from patients with VEO-IBD who commenced adalimumab therapy before the age of 6 years. RESULTS: Seventy-eight patients (37 Crohn's disease, 26 ulcerative colitis, and 15 with IBD-unclassified) were included. Median age of IBD onset was 2.6 (1.3-4.1) years, with 30 (38.5%) patients diagnosed at age <2 years. Median age at adalimumab initiation was 4.2 (2.8-5.1) years. Adalimumab was used as second-line biologic therapy in 45 (57.7%) patients after infliximab. The median time to last follow-up was 63 (22-124) weeks. Significant improvement in clinical scores, CRP, fecal calprotectin, and weight Z-score were observed by Week 52. Adalimumab durability rates were 61.9%, 48.1%, and 35.6% after 1, 2, and 3 years, respectively. Drug discontinuation rates were not dependent on IBD type, age, prior anti-TNF exposure, or concomitant immunomodulatory treatment. Four (5.1%) patients developed serious infections, including 1 patient with TTC7A deficiency who died following adenovirus sepsis. CONCLUSION: Adalimumab therapy is a viable therapeutic option in patients with VEO-IBD with an acceptable safety profile.
The study evaluates adalimumab’s effectiveness and safety in very early-onset inflammatory bowel disease patients. Significant improvements in clinical scores and inflammation markers were observed by Week 52, with durability rates declining over time. Adalimumab had an acceptable safety profile.
Department of NEUROFARBA University of Florence 50121 Florence Italy
Department of Paediatric Gastroenterology Southampton Children's Hospital Southampton SO16 6YD UK
Department of Paediatrics University College Dublin Dublin 4 Ireland
Department of Paediatrics University of Medicine and Health Sciences RCSI Dublin 2 Ireland
Department of Pediatrics Paracelsus Medical University Salzburg 5020 Austria
Department of Pediatrics University of Oxford Oxford OX3 9DU UK
Faculty of Medicine and Health Sciences Tel Aviv University Tel Aviv 6997801 Israel
Faculty of Medicine The Hebrew University of Jerusalem Jerusalem 9190500 Israel
Gastroenterology and Nutrition Unit Meyer Children's Hospital IRCCS 50139 Florence Italy
General Pediatrics and Pediatric Gastroenterology Justus Liebig University 35390 Giessen Germany
Institute for Maternal and Child Health IRCCS Burlo Garofolo 34137 Trieste Italy
National Institute for Health and Care Research Oxford Biomedical Research Centre Oxford OX3 9DU UK
Paediatric Gastroenterology Royal London Children's Hospital Barts Health NHS Trust London E1 1FR UK
Pediatric Gastroenterology and Nutrition Unit Miguel Servet Hospital 50009 Zaragoza Spain
Pediatric Gastroenterology and Nutrition Unit Sachs' Children's Hospital 171 77 Stockholm Sweden
Translational Gastroenterology Unit University of Oxford Oxford OX1 2JD UK
References provided by Crossref.org
