{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,13]],"date-time":"2026-07-13T12:53:56Z","timestamp":1783947236182,"version":"3.55.0"},"reference-count":22,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T00:00:00Z","timestamp":1694736000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2023,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Cystic fibrosis (CF) is the most frequent recessive autosomal disorder in the Caucasian population. It is caused by mutations that result in a deficient or dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Among CFTR modulators, potentiator compounds increase channel opening, whereas corrector compounds increase CFTR quantity in the cell surface.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>To report real\u2010life effects of a generic formulation of lumacaftor\u2010ivacaftor use in patients with CF homozygous for the Phe508del CFTR mutation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients and Methods<\/jats:title><jats:p>Clinical variables (body mass index [BMI], pulmonary exacerbations, sweat test, and pulmonary function) were analyzed in 30 CF patients homozygous for the Phe508del CFTR mutation, treated with lumacaftor\u2010ivacaftor for 12 months, at the Respiratory Center of Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez. These clinical variables were compared with those before the use of modulators.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 30 patients with CF homozygous for the Phe508del CFTR mutation receiving lumacaftor\u2010ivacaftor therapy were included in this study. The median (interquartile range [IQR]) age at the start of treatment was 10.79 (7.08\u201314.05) years. Nineteen patients were male. Before treatment, median (IQR) sweat chloride concentration was 80 (72\u201392) mEq\/L, and it had decreased to 74 (68\u201378) mEq\/L (<jats:italic>p<\/jats:italic>\u2009=\u2009.05) 12 months after treatment. Median (IQR) BMI z\u2010score improved from \u22120.33 (\u22120.86 to 0.21) to \u22120.13 (\u22120.66 to 0.54) (<jats:italic>p<\/jats:italic>\u2009=\u2009.003). A spirometry was performed in 28 of 30 patients. Median (IQR) ppFEV<jats:sub>1<\/jats:sub> was 83.5 (71\u201391) before treatment and 86.5 (67\u2013103) after treatment (<jats:italic>p<\/jats:italic>\u2009=\u2009.38), 73.3% of patients referred decreased sputum production and 40% reported improvement in their dyspnea at 12 months. Severe pulmonary exacerbations significantly decreased from 60% in the year before treatment, to 30% at 12 months after treatment (<jats:italic>p<\/jats:italic>\u2009=\u2009.037); 13 patients showed an improvement in their exacerbation rates, 2 showed an increased rate, and 15 showed no change.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The use of a generic formulation of lumacaftor\u2010ivacaftor in patients homozygous for the Phe508del CFTR mutation was associated with improvement in nutritional status and respiratory symptoms, and a significant reduction in severe pulmonary exacerbations.<\/jats:p><\/jats:sec>","DOI":"10.1002\/ppul.26690","type":"journal-article","created":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T12:06:35Z","timestamp":1694779595000},"page":"3560-3565","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Real\u2010life experience with a generic formulation of lumacaftor\u2010ivacaftor in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation"],"prefix":"10.1002","volume":"58","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7835-9713","authenticated-orcid":false,"given":"Alejandro","family":"Teper","sequence":"first","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvina","family":"Lubovich","sequence":"additional","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viviana","family":"Rodr\u00edguez","sequence":"additional","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvina","family":"Zaragoza","sequence":"additional","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ezequiel","family":"Rodr\u00edguez","sequence":"additional","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Facundo Garc\u00eda","family":"Bournissen","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Schulich School of Medicine and Dentistry Western University London Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,9,15]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)00576-6"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1694021"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.00592-2015"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1183\/09031936.00196314"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1105185"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1409547"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1091\/mbc.e14-04-0935"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF02185761"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(16)30121-7"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2018.12.011"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.03.003"},{"key":"e_1_2_10_13_1","doi-asserted-by":"crossref","unstructured":"MurerC HuberLC KurowskiT HirtA et al.C. 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