{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T03:17:24Z","timestamp":1785554244697,"version":"3.56.0"},"reference-count":34,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2024,6,13]],"date-time":"2024-06-13T00:00:00Z","timestamp":1718236800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2024,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Elexacaftor\u2010tezacaftor\u2010ivacaftor (ETI) is a highly effective cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulating therapy for people with CF and at least one F508del variant. However, there is limited data about the safety and efficacy of this therapy in pediatric populations and in real\u2010world settings. This study aimed at evaluating the effectiveness, tolerability, and safety of ETI in children with CF.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a prospective observational study including all children aged 6\u201311 years who initiated ETI therapy between October 2022 and March 2023 at the Pediatric CF Center of Milan (Italy). Study outcomes included changes in sweat chloride concentration, FEV<jats:sub>1<\/jats:sub>, LCI<jats:sub>2.5<\/jats:sub>, body mass index\u00a0(BMI), tolerance, and safety. Mean changes in study outcomes from baseline through 24 weeks were estimated using mixed\u2010effects regression models.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The study included 34 children with CF (median age: 8.3 years). At Week 12, we observed an average decrease in LCI<jats:sub>2.5<\/jats:sub> of 2.3 units (95% confidence interval [CI]: \u22123.1; \u22121.5). At Week 24, sweat chloride concentration decreased by 63\u2009mEq\/L (95% CI: \u221269; \u221258), FEV<jats:sub>1<\/jats:sub> increased by 8.8 percentage point (95% CI: 3.7; 13.9) and BMI increased by 0.15 standard deviation scores (95% CI: 0.04; 0.25). Skin rashes appeared in 6 patients which spontaneously resolved within a few days. One month after treatment initiation, one patient experienced an elevation in liver function test results, which subsequently decreased during follow\u2010up visits without necessitating discontinuation of therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Our data indicate that ETI therapy is well tolerated by children with CF and is effective in improving signs of lung function abnormalities from early childhood.<\/jats:p><\/jats:sec>","DOI":"10.1002\/ppul.27125","type":"journal-article","created":{"date-parts":[[2024,6,13]],"date-time":"2024-06-13T12:55:29Z","timestamp":1718283329000},"page":"2792-2799","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Effectiveness and safety of elexacaftor\/tezacaftor\/ivacaftor treatment in children aged 6\u201011 years with cystic fibrosis in a real\u2010world setting"],"prefix":"10.1002","volume":"59","author":[{"given":"Valeria","family":"Dacc\u00f2","sequence":"first","affiliation":[{"name":"Pediatric Department, Cystic Fibrosis Pediatric Center Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Rosazza","sequence":"additional","affiliation":[{"name":"Pediatric Department, Cystic Fibrosis Pediatric Center Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Mariani","sequence":"additional","affiliation":[{"name":"Pediatric Department, Cystic Fibrosis Pediatric Center Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmela","family":"Rizza","sequence":"additional","affiliation":[{"name":"Pediatric Department, Cystic Fibrosis Pediatric Center Fondazione 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