{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T11:49:26Z","timestamp":1782474566098,"version":"3.54.5"},"reference-count":41,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,6,10]],"date-time":"2026-06-10T00:00:00Z","timestamp":1781049600000},"content-version":"vor","delay-in-days":9,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2026,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Cystic fibrosis (CF) is a progressive, life\u2010shortening disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (\n                      <jats:italic>CFTR)<\/jats:italic>\n                      gene. CFTR modulator therapies targeting the common\n                      <jats:italic>Phe508del<\/jats:italic>\n                      mutation have been introduced in clinical practice. This study evaluates the real\u2010world effects of 1 year of dual CFTR modulator therapy \u2010 lumacaftor\/ivacaftor or tezacaftor\/ivacaftor \u2010 in children homozygous for\n                      <jats:italic>Phe508del<\/jats:italic>\n                      .\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      A retrospective single\u2010center (Belgium) study was conducted in children with CF aged 2\u201318 years who initiated dual modulator therapy. Clinical data from 1 year before and after treatment initiation were analyzed. Outcomes included changes in sweat chloride concentration (SCC), lung function (percent predicted forced expiratory volume in 1\u2009s (ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      )), frequency of exacerbations and hospitalizations, sputum microbiology, nutritional status (body mass index (BMI) z\u2010score), liver function, vitamin levels, and ophthalmologic status.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Fifty\u2010four children (mean age 8.5 years) were included. After 1 year, SCC decreased significantly with a mean of 14\u2009mmol\/L (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001). Lung function showed a modest improvement with a mean increase of 2.3% in ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      (range: +16% to \u221215%,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.023). No significant changes were observed in exacerbation or hospitalization rates, nor in sputum microbiology. Nutritional status and liver function remained stable, though vitamin A levels increased significantly. The therapy was generally well\u2010tolerated.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>\n                      This real\u2010world study demonstrates that dual CFTR modulators in children homozygous for\n                      <jats:italic>Phe508del<\/jats:italic>\n                      result in a significant reduction in SCC and modest improvements in ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      . These findings support the efficacy and safety of dual modulator therapy in pediatric CF care.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ppul.71701","type":"journal-article","created":{"date-parts":[[2026,6,11]],"date-time":"2026-06-11T06:17:43Z","timestamp":1781158663000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real\u2010World Clinical Outcomes of Lumacaftor\u2010Ivacaftor or Tezacaftor\u2010Ivacaftor Therapy in Children With Cystic Fibrosis Homozygous for\n                    <i>Phe508del<\/i>"],"prefix":"10.1002","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0009-0009-1031-2765","authenticated-orcid":false,"given":"Nele","family":"Aerssens","sequence":"first","affiliation":[{"name":"Pediatrics Department UZ Leuven Leuven 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