ArticleInflammatory bowel diseases2024
Efficacy and Tolerance of Thalidomide in Patients With Very Early Onset Inflammatory Bowel Disease.
Article in Inflammatory bowel diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed, 10 citations in OpenAlex.
- Cumulative thalidomide exposure, peripheral neuropathy, and clinical outcomes in pediatric and very early-onset inflammatory bowel disease: a retrospective cohort study.Clinics (Sao Paulo, Brazil) · 2026Article
- A Comprehensive Review of IL-10 and IL-10 Receptor Deficiencies: From Basic Science to Clinical Bedside.Clinical reviews in allergy & immunology · 2026Review
- Clinical features and prognostic analysis of very early-onset inflammatory bowel disease.BMC pediatrics · 2026Article
- Review on advances in small molecule inhibitors on the treatment of inflammatory bowel disease.Molecular biology reports · 2026Review
- Thalidomide-mediated immunomodulation in a patient withFrontiers in immunology · 2026Article
- Renaissance of Phoenix Drug Thalidomide-New Insights into Practical Clinical Application and Optimization Strategies for Managing Adverse Effects in Digestive Diseases.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Clinical and Phenotypic Characteristics of Early-Onset Inflammatory Bowel Disease: A Five-Year Observational Study.Children (Basel, Switzerland) · 2025Article
- Thalidomide mitigates Crohn's disease colitis by modulating gut microbiota, metabolites, and regulatory T cell immunity.Journal of pharmaceutical analysis · 2025Article
- Clinical characteristics and treatment strategies for A20 haploinsufficiency in Japan: a national epidemiological survey.Frontiers in immunology · 2025Article
Corrections and comments
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Authors and funding
13 authors at 9 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundFew drugs have been studied for patients with very early onset inflammatory bowel disease (VEOIBD). This study aimed to evaluate the efficacy and tolerance of thalidomide in children with VEOIBD compared with children with pediatric-onset IBD (pIBD).
methodsA retrospective cohort study with a control group was conducted. Propensity score 1:1 matching was used to identify control subjects. The treatment persistence; the causes of drug withdrawal; the rate of clinical remission and mucosal healing at 1, 2, and 3 years; and adverse events (AEs) were evaluated in children with VEOIBD treated with thalidomide and compared with children with pIBD.
resultsThirty-nine courses of treatment with thalidomide in VEOIBD and pIBD patients were compared. The treatment persistence at 1, 2, and 3 years was 68.2% (95% confidence interval [CI], 50.8%-80.6%), 57.0% (95% CI, 39.6%-71.1%), and 50.9% (95% CI, 33.7%-65.8%) for VEOIBD patients and 81.7% (95% CI, 65.3%-90.9%), 60.0% (95% CI, 41.7%-74.3%) and 33.0% (95% CI, 17.4%-49.5%) for pIBD patients, respectively (P = .12). A significantly higher proportion of VEOIBD patients discontinued therapy due to lack of efficacy (48.2% vs 17.2%; P = .03), while AEs were the main reason for discontinuation in pIBD patients. Clinical remission and mucosal healing rates did not significantly differ between VEOIBD and pIBD patients. A significatively lower number of VEOIBD patients experienced AEs compared with pIBD patients (14 [35.9%] vs 30 [76.9%]; P = .0005).
conclusionsThalidomide is an effective and tolerated treatment in children with VEOIBD. Discontinuation due to lack of efficacy is more frequent, but AEs are less common than in children with pIBD.
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