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It is not known whether LUM\/IVA use in children can impact the progression of structural lung disease. We sought to determine the real-world impact of LUM\/IVA on lung structure and function in children aged 6\u201311 years.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      This real-world observational cohort study was conducted across four paediatric sites in Ireland over 24-months using spirometry-controlled CT scores and LCI as primary outcome measures. Children commencing LUM-\/IVA as part of routine care were included. CT scans were manually scored with the PRAGMA CF scoring system and analysed using the automated bronchus-artery (BA) method. Secondary outcome measures included rate of change of ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      , nutritional indices and exacerbations requiring hospitalisation.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Seventy-one participants were recruited to the study, 31 of whom had spirometry-controlled CT performed at baseline, and after one year and two years of LUM\/IVA treatment. At two years there was a reduction from baseline in trapped air scores (0.13 to 0.07, p\u2009=\u20090.016), but an increase from baseline in the % bronchiectasis score (0.84 to 1.23, p\u2009=\u20090.007). There was no change in overall % disease score (2.78 to 2.25, p\u2009=\u20090.138). Airway lumen to pulmonary artery ratios (A\n                      <jats:sub>lumen<\/jats:sub>\n                      A ratio) were abnormal at baseline and worsened over the course of the study. In 28 participants, the mean annual change from baseline LCI\n                      <jats:sub>2.5<\/jats:sub>\n                      (-0.055 (-0.61 to 0.50), p\u2009=\u20090.85) measurements over two years were not significant. Improvements from baseline in weight (0.10 (0.06 to 0.15, p\u2009&lt;\u20090.0001), height (0.05 (0.02 to 0.09), p\u2009=\u20090.002) and BMI (0.09 (0.03 to 0.15) p\u2009=\u20090.005) z-scores were seen with LUM\/IVA treatment. The mean annual change from baseline ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      (-2.45 (-4.44 to 2.54), p\u2009=\u20090.66) measurements over two years were not significant.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>In a real-world setting, the use of LUM\/IVA over two years in children with CF aged 6\u201311 resulted in improvements in air trapping on CT but worsening in bronchiectasis scores. Our results suggest that LUM\/IVA use in this age group improves air trapping but does not prevent progression of bronchiectasis over two years of treatment.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s12931-023-02497-0","type":"journal-article","created":{"date-parts":[[2023,8,11]],"date-time":"2023-08-11T06:01:57Z","timestamp":1691733717000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6\u201311 with cystic fibrosis in a real-world setting"],"prefix":"10.1186","volume":"24","author":[{"given":"Paul","family":"McNally","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry","family":"Linnane","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Williamson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Basil","family":"Elnazir","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Short","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clare","family":"Saunders","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Kirwan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rea","family":"David","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariette P. 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ERJ Open Res, 2021. 7(1).","DOI":"10.1183\/23120541.00203-2020"}],"container-title":["Respiratory Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s12931-023-02497-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s12931-023-02497-0\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s12931-023-02497-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,18]],"date-time":"2023-11-18T22:22:37Z","timestamp":1700346157000},"score":1,"resource":{"primary":{"URL":"https:\/\/respiratory-research.biomedcentral.com\/articles\/10.1186\/s12931-023-02497-0"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8,11]]},"references-count":48,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2023,12]]}},"alternative-id":["2497"],"URL":"https:\/\/doi.org\/10.1186\/s12931-023-02497-0","relation":{"has-preprint":[{"id-type":"doi","id":"10.21203\/rs.3.rs-2582978\/v1","asserted-by":"object"}]},"ISSN":["1465-993X"],"issn-type":[{"value":"1465-993X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8,11]]},"assertion":[{"value":"13 February 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"21 July 2023","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"11 August 2023","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The research ethics committee at Children\u2019s Health Ireland (CHI) (Reference number: GEN\/620\/18) approved the study and informed consent and assent was obtained.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Professor Jane Davies is supported by the National Institutes of Health Research through the Imperial Biomedical Research Centre, the Royal Brompton Clinical Research Facility and a Senior Investigator Award. The remaining authors declare no competing interests.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"199"}}