{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T16:34:30Z","timestamp":1788885270719,"version":"build-2803163510"},"reference-count":22,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T00:00:00Z","timestamp":1676592000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,1,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Background<\/jats:title>\n                  <jats:p>Few 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).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>A 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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Thirty-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).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusions<\/jats:title>\n                  <jats:p>Thalidomide 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.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1093\/ibd\/izad018","type":"journal-article","created":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T13:50:24Z","timestamp":1676641824000},"page":"20-28","source":"Crossref","is-referenced-by-count":12,"title":["Efficacy and Tolerance of Thalidomide in Patients With Very Early Onset Inflammatory Bowel Disease"],"prefix":"10.1093","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5249-8248","authenticated-orcid":false,"given":"Matteo","family":"Bramuzzo","sequence":"first","affiliation":[{"name":"Institute for Maternal and Child Health, IRCCS Burlo Garofolo , Trieste , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabiola","family":"Giudici","sequence":"additional","affiliation":[{"name":"Bureau de biostatistique et d\u2019\u00e9pidemiologie, Gustave Roussy and Universit\u00e9 Paris-Saclay , Paris , France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Serena","family":"Arrigo","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology and Endoscopy, IRCCS Istituto Giannina Gaslini, Genoa , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1056-4400","authenticated-orcid":false,"given":"Paolo","family":"Lionetti","sequence":"additional","affiliation":[{"name":"Department NEUROFARBA, Meyer Children\u2019s Hospital, University of Florence , Florence , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanna","family":"Zuin","sequence":"additional","affiliation":[{"name":"Pediatrics , Fondazione IRCCS San Gerardo dei Tintori, Monza , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio","family":"Romano","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology and Cystic Fibrosis Unit, Department of Human Pathology in Adulthood and Childhood G. Barresi, University of Messina , Messina , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4896-1842","authenticated-orcid":false,"given":"Francesco","family":"Graziano","sequence":"additional","affiliation":[{"name":"Pediatric Unit, Villa Sofia Cervello Hospital , Palermo , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simona","family":"Faraci","sequence":"additional","affiliation":[{"name":"Digestive Endoscopy and Surgery Unit, Bambino Ges\u00f9 Children\u2019s Hospital , Rome , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrizia","family":"Alvisi","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology Unit, Maggiore Hospital, Bologna , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Signa","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology and Endoscopy, IRCCS Istituto Giannina Gaslini, Genoa , 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