{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T13:39:53Z","timestamp":1780407593365,"version":"3.54.1"},"reference-count":28,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2026,2,17]],"date-time":"2026-02-17T00:00:00Z","timestamp":1771286400000},"content-version":"vor","delay-in-days":16,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"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":[[2026,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Cystic Fibrosis Transmembrane Conductance Regulator\n                      <jats:italic>(CFTR)<\/jats:italic>\n                      modulators revolutionized the care of people with Cystic fibrosis (pwCF) by addressing the underlying genetic defect rather than merely managing symptoms. Elexacaftor\/Tezacaftor\/Ivacaftor (ETI) has shown significant clinical benefits in pwCF carrying specific\n                      <jats:italic>CFTR<\/jats:italic>\n                      mutations; however, data on its safety and efficacy in young children with CF (ycwCF), aged 2\u20136 years, remain sparse.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      We conducted a real\u2010world, retrospective study that included all Israeli ycwCF who were genetically eligible for and treated with ETI. Body mass index (BMI) z\u2010score, forced expiratory volume in one second (FEV\n                      <jats:sub>1<\/jats:sub>\n                      ) percent predicted, rate of pulmonary exacerbations (PEx), sweat conductivity levels, bacterial sputum isolates, clinic visits frequency, and sputum samples collected before and after ETI initiation were evaluated. Additionally, adverse effects were assessed.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Sixteen ycwCF, aged 2\u20136 years, received ETI therapy for a mean duration of 13 months (range 4\u201325 months), including three pancreatic\u2010sufficient patients. Following ETI initiation, significant improvements were observed. BMI z\u2010scores increased from \u22120.37\u2009\u00b1\u20091.32 to \u22120.01\u2009\u00b1\u20091.14 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.005), and in the subset of patients tested (7\/16), FEV\n                      <jats:sub>1<\/jats:sub>\n                      improved from 91\u2009\u00b1\u200912 to 113\u2009\u00b1\u200918 percent predicted (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.018). The rate of PEx decreased from 0.7\u202f\u2009\u00b1\u2009\u202f1.1 to 0.2\u202f\u2009\u00b1\u2009\u202f0.4 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.058) and sweat conductivity levels dropped significantly from 112\u202f\u2009\u00b1\u2009\u202f38 to 48\u202f\u2009\u00b1\u2009\u202f30\u2009mmol\/L (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.002). There was a marked reduction in bacterial colonization in sputum:\n                      <jats:italic>Pseudomonas aeruginosa<\/jats:italic>\n                      declined from 9\/16 to 2\/14 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.04), and\n                      <jats:italic>Staphylococcus aureus<\/jats:italic>\n                      from 12\/16 to 2\/14 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.01). Notably, the total number of sputum cultures sent also decreased by 35%, from 116 before ETI to 75 after treatment initiation. This reduction paralleled decreased CF clinic visits, from 7.3\u202f\u2009\u00b1\u2009\u202f3 per year to 4.7\u202f\u2009\u00b1\u2009\u202f3 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.001). Reported adverse effects were minimal, and no treatment discontinuations were required.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>These real\u2010world findings support the safety and effectiveness of ETI in ycwCF. Robust, long\u2010term studies involving larger populations are essential to confirm these results.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ppul.71523","type":"journal-article","created":{"date-parts":[[2026,2,18]],"date-time":"2026-02-18T07:15:56Z","timestamp":1771398956000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Real\u2010World Impact of Elexacaftor\/Tezacaftor\/Ivacaftor in Israeli Children Aged 2\u20136 With Cystic Fibrosis"],"prefix":"10.1002","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0427-571X","authenticated-orcid":false,"given":"Adi","family":"Dagan","sequence":"first","affiliation":[{"name":"Pediatric Pulmonary Unit and CF Center, The Edmond and Lili Safra Children's Hospital, Chaim Sheba Medical Center, Tel Hashomer, and Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamad","family":"Hadhud","sequence":"additional","affiliation":[{"name":"Pediatric Pulmonary Unit and CF Center, Hadassah Medical Center and Faculty of Medicine Hebrew University of Jerusalem Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ori","family":"Efrati","sequence":"additional","affiliation":[{"name":"Pediatric Pulmonary Unit and CF Center, The Edmond and Lili Safra Children's Hospital, Chaim Sheba Medical Center, Tel Hashomer, and Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon Sarah","family":"Cahanovitc","sequence":"additional","affiliation":[{"name":"Pulmonary Institute and Graub CF Center, Schneider Children's Medical Center of Israel, Petah Tikva and Faculty of Medical and Health Sciences Tel\u2010Aviv University Tel\u2010Aviv 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