{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T06:14:27Z","timestamp":1787465667083,"version":"build-2736575974"},"reference-count":32,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2025,10,3]],"date-time":"2025-10-03T00:00:00Z","timestamp":1759449600000},"content-version":"vor","delay-in-days":2,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Translational Sci"],"published-print":{"date-parts":[[2025,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Elexacaftor\/Tezacaftor\/Ivacaftor (ETI) therapy has significantly improved clinical outcomes in people with cystic fibrosis (PwCF) carrying at least one Phe508del CFTR mutation. However, real\u2010world evidence on the safety and effectiveness of ETI in pediatric populations remains limited, particularly in children with more severe disease phenotypes who are often excluded from clinical trials. We analyzed clinical, functional, and microbiological data from pediatric CF patients aged 6\u201317\u2009years treated with ETI between October 2022 and March 2024. Lung function (ppFEV\n                    <jats:sub>1<\/jats:sub>\n                    , ppFVC), nutritional status (BMI, BMI\n                    <jats:italic>z<\/jats:italic>\n                    \u2010score), sweat chloride concentration (SwCl), quality of life (CFQ\u2010R), pulmonary exacerbations (PEx), and airway pathogens were assessed at baseline and after 6\u201312\u2009months. Adverse events (AEs) and therapy discontinuations were also recorded. Twenty\u2010four patients (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200910 aged 6\u201311\u2009years;\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200914 aged 12\u201317\u2009years) were included. At 12\u2009months, mean ppFEV\n                    <jats:sub>1<\/jats:sub>\n                    increased by 15% (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.013), BMI by 2.4\u2009kg\/m\n                    <jats:sup>2<\/jats:sup>\n                    (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.16), BMI weight z\u2010score by 0.33 (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.63), and height\n                    <jats:italic>z<\/jats:italic>\n                    \u2010score by \u22120.33 (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.72), SwCl decreased by 46\u2009mmol\/L (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001), and CFQ\u2010R respiratory domain improved by 14 points (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001). PEx rates decreased by 27.6% after 12\u2009months. Reductions in airway pathogen prevalence, particularly\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>Staphylococcus aureus<\/jats:italic>\n                    <\/jats:styled-content>\n                    and\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>Pseudomonas aeruginosa<\/jats:italic>\n                    <\/jats:styled-content>\n                    , were observed. AEs occurred in 14.8% (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20094) of patients, were mild\u2010to\u2010moderate, and resolved with dose reduction. ETI therapy was associated with marked improvements in lung function, nutritional status, and quality of life, along with reductions in PEx and airway pathogens, in a real\u2010world pediatric CF cohort. These findings support ETI use in this population, with careful AE monitoring and dose adjustment when needed.\n                  <\/jats:p>","DOI":"10.1111\/cts.70373","type":"journal-article","created":{"date-parts":[[2025,10,3]],"date-time":"2025-10-03T07:33:33Z","timestamp":1759476813000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Real\u2010World Evaluation of Outcomes and Safety of Elexacaftor\/Tezacaftor\/Ivacaftor in Pediatric Patients With Cystic Fibrosis: A Retrospective Study"],"prefix":"10.1111","volume":"18","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0809-5118","authenticated-orcid":false,"given":"Nicola","family":"Perrotta","sequence":"first","affiliation":[{"name":"Department of Physiology and Pharmacology \u201cV. Erspamer\u201d Sapienza University  Rome Italy"},{"name":"Pharmacy Unit AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luigi Angelo","family":"Fiorito","sequence":"additional","affiliation":[{"name":"Department of Physiology and Pharmacology \u201cV. Erspamer\u201d Sapienza University  Rome Italy"},{"name":"Pharmacy Unit AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rossella","family":"Gentile","sequence":"additional","affiliation":[{"name":"Department of Physiology and Pharmacology \u201cV. Erspamer\u201d Sapienza University  Rome Italy"},{"name":"Pharmacy Unit AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberta","family":"Vescovo","sequence":"additional","affiliation":[{"name":"Department of Chemistry and Technology of Drugs Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfonso","family":"Piciocchi","sequence":"additional","affiliation":[{"name":"Biostatistics Unit GIMEMA Foundation  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrizia","family":"Troiani","sequence":"additional","affiliation":[{"name":"Regional Cystic Fibrosis Center AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Poscia","sequence":"additional","affiliation":[{"name":"Clinical Research Unit AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"},{"name":"Interdepartmental Center for Rare Diseases AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Cimino","sequence":"additional","affiliation":[{"name":"Regional Cystic Fibrosis Center AOU Policlinico Umberto I \u2013 Sapienza University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,10,3]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-016-2391-y"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/jpids\/piac052"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.2147\/JEP.S255377"},{"key":"e_1_2_12_5_1","unstructured":"Gazzetta Ufficiale \u201cDETERMINA 1\u00b0 luglio 2021. Riclassificazione del medicinale per uso umano \u00abKaftrio\u00bb ai sensi dell'articolo 8 comma 10 della legge 24 dicembre 1993 n. 537 \u201d(Determina n. DG\/784\/2021)."},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32597-8"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908639"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202102-0509OC"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202202-0392OC"},{"key":"e_1_2_12_10_1","unstructured":"DETERMINA \u201c26 settembre 2022 Regime di rimborsabilita' e prezzo a seguito di nuove indicazioni terapeutiche del medicinale per uso umano \u00abKaftrio\u00bb \u201d(Determina n. 680\/2022) (22A05579) (GU n.227 del 28\u20109\u20102022)."},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.3390\/cells11111769"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2023.1176815"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1183\/20734735.012717"},{"key":"e_1_2_12_14_1","volume-title":"R: A Language and Environment for Statistical Computing","author":"R Core Team","year":"2022"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.3390\/ph16030410"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(21)00454-9"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.03079-2020"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.70083"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1186\/s40348-025-00194-0"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.25980"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.27125"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.heliyon.2024.e28508"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1709846"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1409547"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.2174\/1573396320666230912103056"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.3121\/cmr.2019.1476"},{"key":"e_1_2_12_27_1","first-page":"290","article-title":"Loss of Cftr Function in Neurons Results in Poor Growth and Possible Endocrine Dysfunction","volume":"45","author":"Hodges C. 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