{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T11:49:32Z","timestamp":1782474572425,"version":"3.54.5"},"reference-count":39,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,6,16]],"date-time":"2026-06-16T00:00:00Z","timestamp":1781568000000},"content-version":"vor","delay-in-days":15,"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>Introduction<\/jats:title>\n                    <jats:p>Elexacaftor\/Tezacaftor\/Ivacaftor (ETI) has revolutionized cystic fibrosis (CF) care. In Argentina, the absence of patent restrictions allowed the production of a generic ETI formulation (ETIgf), significantly reducing costs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>This study evaluates the real\u2010world clinical impact of ETIgf in people with CF (pwCF).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      This prospective, single\u2010center observational study in Buenos Aires, Argentina, included 71 pwCF (\u2265\u20096 years) with responsive variants. Participants were categorized as: Group 1 (modulator\u2010na\u00efve) or Group 2 (previously on lumacaftor\/ivacaftor). Clinical outcomes, including ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      , LCI\n                      <jats:sub>2.5<\/jats:sub>\n                      , BMI\n                      <jats:italic>z<\/jats:italic>\n                      \u2010score, CFQ\u2010R, sweat chloride concentration (SCC), fecal elastase levels, liver enzymes and pulmonary exacerbations (PEx), were assessed over 12 months following the initiation of ETIgf.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Group 1 included 43 patients, and Group 2 included 28 patients (median age: 10.9 and 10.6 years, respectively). The mean percentage change from baseline after 12 months of treatment for Group 1 and Group 2 was: ppFEV\n                      <jats:sub>1<\/jats:sub>\n                      +20.8 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01) and +18.5 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01); LCI\n                      <jats:sub>2.5<\/jats:sub>\n                      \u201313.3 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01) and \u201311.2 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05); CFQ\u2010R\u2009+\u200966.1 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01) and +30.4 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01); and SCC \u201353.1 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01) and \u201358.2 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01), respectively. BMI\n                      <jats:italic>z<\/jats:italic>\n                      \u2010score increased by 0.5 in Group 1 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001), while Group 2 showed a non\u2010significant increase. Pulmonary exacerbations were reduced by 95% in both groups. No changes were observed in fecal elastase levels. Adverse events were mild: five patients experienced elevated transaminase levels and three presented with a rash.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>ETIgf demonstrates robust clinical efficacy and safety, mirroring results reported for the originator. These findings highlight the viability of affordable generic modulators in expanding global access to life\u2010changing therapy.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ppul.71706","type":"journal-article","created":{"date-parts":[[2026,6,16]],"date-time":"2026-06-16T07:55:42Z","timestamp":1781596542000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real\u2010Life Experience With a Generic Formulation of Elexacaftor\/Tezacaftor\/Ivacaftor in Patients With Cystic Fibrosis With Responding Variants"],"prefix":"10.1002","volume":"61","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 Aut\u00f3noma 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 Aut\u00f3noma 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 Aut\u00f3noma 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 Aut\u00f3noma de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ezequiel","family":"Rodr\u00edguez Novoa","sequence":"additional","affiliation":[{"name":"Division of Respiratory Hospital de Ni\u00f1os Ricardo Guti\u00e9rrez Ciudad Aut\u00f3noma de Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Facundo","family":"Garc\u00eda Bournissen","sequence":"additional","affiliation":[{"name":"Division of Pediatric Clinical Pharmacology, Department of Paediatrics, Schulich School of Medicine and Dentistry Western University London Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,16]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2006.07.004"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijns6010018"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.25130"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.00592-2015"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)00576-6"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1694021"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202301-0021OC"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2025.10.007"},{"key":"e_1_2_10_10_1","unstructured":"ReNaFQ. Fibrosis Qu\u00edstica (Mutaciones) accessed November 4 2025 https:\/\/inerconi.anlis.gob.ar\/shiny\/Reporte-renafq\/."},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908639"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32597-8"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202102-0509OC"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202202-0392OC"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202301-0084OC"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202411-2231OC"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1183\/23120541.00339-2024"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1513\/AnnalsATS.202509-988ED"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1513\/AnnalsATS.202501-103OC"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.04.007"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26954"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2025.02.010"},{"key":"e_1_2_10_23_1","unstructured":"Law 27552 Honorable Congress of the Argentine Nation| Argentina.gob.ar. accessed November 4 2025 https:\/\/www.argentina.gob.ar\/normativa\/nacional\/ley-27552-340915\/normas-modifican."},{"key":"e_1_2_10_24_1","unstructured":"Trixacar product information. Droguerie Phenica accessed November 4 2025 https:\/\/www.pheniciagroup.com\/trixacar."},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26690"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199409083311003"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2020.12.009"},{"key":"e_1_2_10_28_1","first-page":"557","volume-title":"Clinical Nutrition","author":"Turck D.","year":"2016"},{"key":"e_1_2_10_29_1","unstructured":"WHO \u201cChild Growth Standards: Body mass index\u2010for\u2010age (BMI\u2010for\u2010age)Z\u2010score Tables \u201d World Health Organization. Available at:https:\/\/www.who.int\/toolkits\/child-growth-standards\/standards\/body-mass-index-for-age-bmi-for-age."},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.prrv.2020.04.003"},{"key":"e_1_2_10_31_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202008-3177OC"},{"key":"e_1_2_10_32_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.71156"},{"key":"e_1_2_10_33_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1697975"},{"key":"e_1_2_10_34_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.200909-1421OC"},{"key":"e_1_2_10_35_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40266-025-01207-3"},{"key":"e_1_2_10_36_1","doi-asserted-by":"publisher","DOI":"10.3389\/fdsfr.2024.1363794"},{"key":"e_1_2_10_37_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2025.42041"},{"key":"e_1_2_10_38_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202108-1986OC"},{"key":"e_1_2_10_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(21)00454-9"},{"key":"e_1_2_10_40_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2025.02.002"}],"container-title":["Pediatric Pulmonology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.71706","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/ppul.71706","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.71706","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T11:31:26Z","timestamp":1782473486000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ppul.71706"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6]]},"references-count":39,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["10.1002\/ppul.71706"],"URL":"https:\/\/doi.org\/10.1002\/ppul.71706","archive":["Portico"],"relation":{},"ISSN":["8755-6863","1099-0496"],"issn-type":[{"value":"8755-6863","type":"print"},{"value":"1099-0496","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,6]]},"assertion":[{"value":"2026-02-17","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-06-08","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-06-16","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e71706"}}