{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,28]],"date-time":"2026-01-28T06:40:20Z","timestamp":1769582420716,"version":"3.49.0"},"reference-count":73,"publisher":"MDPI AG","issue":"2","license":[{"start":{"date-parts":[[2023,2,9]],"date-time":"2023-02-09T00:00:00Z","timestamp":1675900800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Background: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators represent targeted therapies directly acting on the CFTR channel. The triple therapy Elexacaftor\/Tezacaftor\/Ivacaftor (ELX\/TEZ\/IVA) has been demonstrated to improve lung function and quality of life in cystic fibrosis (CF) patients. However, the effects of ELX\/TEZ\/IVA on sleep-disordered breathing (SDB) and respiratory muscle strength are poorly studied. The aim of this study was to assess the effects of ELX\/TEZ\/IVA in patients with CF and severe lung disease on cardiorespiratory polygraphy parameters, maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP) measures. Methods: patients with CF aged \u2265 12 who started treatment in a compassionate use program were retrospectively studied through the evaluation of nocturnal cardiorespiratory polygraphy parameters, MIP and MEP; and six-minute walk test (6MWT) at baseline and at months 3, 6, and 12 of treatment. Results: Nine patients (mean age 30.3 \u00b1 6.5 years) with severe CF (mean baseline ppFEV1 34.6 \u00b1 5.1%) were evaluated. A significant improvement in nocturnal oxygenation measured by mean SpO2 (92.4 vs. 96.4%, p &lt; 0.05), time spent with SpO2 \u2264 90% (\u221212.6, \u221214.6, \u221215.2 min from baseline at months 3, 6, and 12, respectively, p &lt; 0.05), and respiratory rate (RR) was shown, at month 12 and across the time points compared with baseline, as well as in respiratory muscle strength, although only the change in MEP was significant. Conclusions: We provide further evidence on the efficacy of the CFTR modulators ELX\/TEZ\/IVA, adding information about their effect on the respiratory muscles\u2019 performance and cardiorespiratory polygraphy parameters in CF patients with severe lung disease.<\/jats:p>","DOI":"10.3390\/genes14020449","type":"journal-article","created":{"date-parts":[[2023,2,10]],"date-time":"2023-02-10T03:52:49Z","timestamp":1676001169000},"page":"449","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Effects of Elexacaftor\/Tezacaftor\/Ivacaftor on Cardiorespiratory Polygraphy Parameters and Respiratory Muscle Strength in Cystic Fibrosis Patients with Severe Lung Disease"],"prefix":"10.3390","volume":"14","author":[{"given":"Alessandro","family":"Giallongo","sequence":"first","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4291-0195","authenticated-orcid":false,"given":"Giuseppe Fabio","family":"Parisi","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria","family":"Papale","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7664-3083","authenticated-orcid":false,"given":"Sara","family":"Manti","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"},{"name":"Pediatric Unit, Department of Human and Pediatric Pathology \u201cGaetano Barresi\u201d, AOUP G. Martino, University of Messina, Via Consolare Valeria, 1, 98124 Messina, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Enza","family":"Mul\u00e9","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Donatella","family":"Aloisio","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1106-4424","authenticated-orcid":false,"given":"Vito","family":"Terlizzi","sequence":"additional","affiliation":[{"name":"Cystic Fibrosis Regional Reference Center, Department of Pediatric Medicine, Meyer Children\u2019s Hospital IRCCS, 50139 Firenze, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Novella","family":"Rotolo","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2642-6400","authenticated-orcid":false,"given":"Salvatore","family":"Leonardi","sequence":"additional","affiliation":[{"name":"Pediatric Respiratory and Cystic Fibrosis Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,2,9]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene: Cloning and characterization of complementary","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"key":"ref_2","unstructured":"(2022, November 28). 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