{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T03:17:23Z","timestamp":1785554243007,"version":"3.56.0"},"reference-count":32,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2025,3,1]],"date-time":"2025-03-01T00:00:00Z","timestamp":1740787200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,3,1]],"date-time":"2025-03-01T00:00:00Z","timestamp":1740787200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,2,18]],"date-time":"2025-02-18T00:00:00Z","timestamp":1739836800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1TR002733"],"award-info":[{"award-number":["UL1TR002733"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["MCCOY19RO"],"award-info":[{"award-number":["MCCOY19RO"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008747","name":"Center for Clinical and Translational Science, Ohio State University","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008747","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100024158","name":"ARS Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100024158","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006483","name":"AbbVie","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006483","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100001003","name":"Boehringer Ingelheim","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100001003","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016616","name":"Cystic Fibrosis Research Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016616","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Pulmonary Pharmacology & Therapeutics"],"published-print":{"date-parts":[[2025,3]]},"DOI":"10.1016\/j.pupt.2025.102345","type":"journal-article","created":{"date-parts":[[2025,2,18]],"date-time":"2025-02-18T19:17:30Z","timestamp":1739906250000},"page":"102345","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"special_numbering":"C","title":["Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis"],"prefix":"10.1016","volume":"88","author":[{"given":"Shahid","family":"Sheikh","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Holtzlander","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariah","family":"Eisner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney","family":"Gushue","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Palacios","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kavitha","family":"Kotha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sehyr","family":"Imran","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen S.","family":"McCoy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.pupt.2025.102345_bib2","series-title":"Bethesda, MD: Cystic Fibrosis Foundation, 2021 Annual Data Report (cff.Org)","article-title":"Patient registry: annual data report","year":"2021"},{"key":"10.1016\/j.pupt.2025.102345_bib3","unstructured":"http:\/\/www.cff.org\/UploadedFiles\/research\/ClinicalResearch\/PatientRegistryReport\/2012-CFF-Patient-Registry.pdf. opens in new tab)."},{"key":"10.1016\/j.pupt.2025.102345_bib4","doi-asserted-by":"crossref","first-page":"1891","DOI":"10.1016\/S0140-6736(09)60327-5","article-title":"Cystic fibrosis","volume":"373","author":"O'Sullivan","year":"2009","journal-title":"Lancet"},{"key":"10.1016\/j.pupt.2025.102345_bib5","doi-asserted-by":"crossref","first-page":"233","DOI":"10.7326\/M13-0636","article-title":"Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry","volume":"161","author":"MacKenzie","year":"2014","journal-title":"Ann. Intern. Med."},{"key":"10.1016\/j.pupt.2025.102345_bib6","doi-asserted-by":"crossref","first-page":"5242","DOI":"10.1128\/MCB.25.12.5242-5252.2005","article-title":"Most F508del-CFTR is targeted to degradation at an early folding checkpoint and independently of calnexin","volume":"25","author":"Farinha","year":"2005","journal-title":"Mol. Cell Biol."},{"key":"10.1016\/j.pupt.2025.102345_bib7","doi-asserted-by":"crossref","first-page":"8942","DOI":"10.1074\/jbc.M009172200","article-title":"Conformational and temperature-sensitive stability defects of the delta F508 cystic fibrosis transmembrane conductance regulator in post-endoplasmic reticulum compartments","volume":"276","author":"Sharma","year":"2001","journal-title":"J. Biol. Chem."},{"issue":"18","key":"10.1016\/j.pupt.2025.102345_bib8","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa1105185","article-title":"A CFTR potentiator in patients with cystic fibrosis and the G551D mutation","volume":"365","author":"Ramsey","year":"2011","journal-title":"N. Engl. J. Med."},{"issue":"6","key":"10.1016\/j.pupt.2025.102345_bib9","doi-asserted-by":"crossref","first-page":"674","DOI":"10.1016\/j.jcf.2014.09.005","article-title":"Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation","volume":"13","author":"De Boeck","year":"2014","journal-title":"J. Cyst. Fibros."},{"issue":"3","key":"10.1016\/j.pupt.2025.102345_bib10","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1056\/NEJMoa1409547","article-title":"Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR","volume":"373","author":"Wainwright","year":"2015","journal-title":"N. Engl. J. Med."},{"issue":"21","key":"10.1016\/j.pupt.2025.102345_bib11","doi-asserted-by":"crossref","first-page":"2013","DOI":"10.1056\/NEJMoa1709846","article-title":"Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del","volume":"377","author":"Taylor-Cousar","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.pupt.2025.102345_bib12","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1164\/rccm.201608-1754OC","article-title":"Lumacaftor\/ivacaftor in patients aged 6\u201311 years with cystic fibrosis and homozygous for F508del-CFTR","volume":"195","author":"Milla","year":"2017","journal-title":"Am. J. Respir. Crit. Care Med."},{"issue":"19","key":"10.1016\/j.pupt.2025.102345_bib13","doi-asserted-by":"crossref","first-page":"1809","DOI":"10.1056\/NEJMoa1908639","article-title":"Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele","volume":"381","author":"Middleton","year":"2019","journal-title":"N. Engl. J. Med."},{"issue":"10212","key":"10.1016\/j.pupt.2025.102345_bib14","doi-asserted-by":"crossref","first-page":"1940","DOI":"10.1016\/S0140-6736(19)32597-8","article-title":"Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial","volume":"394","author":"Heijerman","year":"2019","journal-title":"Lancet"},{"key":"10.1016\/j.pupt.2025.102345_bib15","doi-asserted-by":"crossref","first-page":"897","DOI":"10.1513\/AnnalsATS.201802-075FR","article-title":"Cystic fibrosis: translating molecular mechanisms into effective therapies","volume":"15","author":"Jennings","year":"2018","journal-title":"Ann Am Thorac Soc"},{"key":"10.1016\/j.pupt.2025.102345_bib16","doi-asserted-by":"crossref","first-page":"7234","DOI":"10.1038\/s41598-019-43652-2","article-title":"A systematic review of the clinical efficacy and safety of CFTR modulators in cystic fibrosis","volume":"9","author":"Habib","year":"2019","journal-title":"Sci. Rep."},{"issue":"12","key":"10.1016\/j.pupt.2025.102345_bib17","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1001\/jamaoto.2023.2701","article-title":"Elexacaftor-tezacaftor-ivacaftor therapy for chronic sinus disease in cystic fibrosis","volume":"149","author":"Sheikh","year":"2023","journal-title":"JAMA Otolaryngol Head Neck Surg"},{"issue":"3","key":"10.1016\/j.pupt.2025.102345_bib18","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1002\/ppul.26261","article-title":"Impact of elexacaftor-tezacaftor-ivacaftor on bacterial colonization and inflammatory responses in cystic fibrosis","volume":"58","author":"Sheikh","year":"2023","journal-title":"Pediatr. Pulmonol."},{"issue":"3","key":"10.1016\/j.pupt.2025.102345_bib19","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1016\/j.jcf.2022.12.009","article-title":"Effect of Elexacaftor\/Tezacaftor\/Ivacaftor on annual rate of lung function decline in people with cystic fibrosis","volume":"22","author":"Lee","year":"2023","journal-title":"J. Cyst. Fibros."},{"issue":"4","key":"10.1016\/j.pupt.2025.102345_bib20","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1016\/j.jcf.2022.12.012","article-title":"Effects of elexacaftor\/tezacaftor\/ivacaftor therapy in children with cystic fibrosis - a comprehensive assessment using lung clearance index, spirometry, and functional and structural lung MRI","volume":"22","author":"Streibel","year":"2023","journal-title":"J. Cyst. Fibros."},{"issue":"4","key":"10.1016\/j.pupt.2025.102345_bib21","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1016\/j.jcf.2023.02.004","article-title":"Elexacaftor\/tezacaftor\/ivacaftor projected survival and long-term health outcomes in people with cystic fibrosis homozygous for F508del","volume":"22","author":"Lopez","year":"2023","journal-title":"J. Cyst. Fibros."},{"issue":"8","key":"10.1016\/j.pupt.2025.102345_bib22","doi-asserted-by":"crossref","first-page":"835","DOI":"10.1136\/thorax-2022-219943","article-title":"Effect of elexacaftor\/tezacaftor\/ivacaftor on airway and systemic inflammation in cystic fibrosis","volume":"78","author":"Casey","year":"2023","journal-title":"Thorax"},{"issue":"4","key":"10.1016\/j.pupt.2025.102345_bib23","doi-asserted-by":"crossref","first-page":"1178","DOI":"10.1002\/ppul.26318","article-title":"Clinical change 2 years from start of elexacaftor-tezacaftor-ivacaftor in severe cystic fibrosis","volume":"58","author":"McCoy","year":"2023","journal-title":"Pediatr. Pulmonol."},{"issue":"3","key":"10.1016\/j.pupt.2025.102345_bib24","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1016\/S2213-2600(21)00454-9","article-title":"Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial","volume":"10","author":"Sutharsan","year":"2022","journal-title":"Lancet Respir. Med."},{"key":"10.1016\/j.pupt.2025.102345_bib25","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1164\/rccm.202108-1986OC","article-title":"Clinical effectiveness of elexacaftor\/tezacaftor\/ivacaftor in people with cystic fibrosis: a clinical trial","volume":"205","author":"Nichols","year":"2022","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.pupt.2025.102345_bib26","doi-asserted-by":"crossref","first-page":"1021","DOI":"10.3390\/jcm11041021","article-title":"Elexacaftor\/tezacaftor\/ivacaftor in patients with cystic fibrosis homozygous for the F508delMutation and advanced lung disease: a 48-week observational study","volume":"11","author":"Carnovale","year":"2022","journal-title":"J. Clin. Med."},{"key":"10.1016\/j.pupt.2025.102345_bib27","doi-asserted-by":"crossref","DOI":"10.1002\/prp2.1015","article-title":"Real-life efficacy and safety of elexacaftor\/tezacaftor\/ivacaftor on severe cystic fibrosis lung disease patients","volume":"10","author":"Kos","year":"2022","journal-title":"Pharmacol. Res. Perspect."},{"key":"10.1016\/j.pupt.2025.102345_bib28","doi-asserted-by":"crossref","DOI":"10.3389\/fphar.2023.1176815","article-title":"Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis","volume":"14","author":"Olivier","year":"2023","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.pupt.2025.102345_bib29","doi-asserted-by":"crossref","DOI":"10.1016\/j.jcf.2022.12.012","article-title":"Effects of elexacaftor\/tezacaftor\/ivacaftor therapy in children with cystic fibrosis - a comprehensive assessment using lung clearance index, spirometry, and functional and structural lung MRI","author":"Streibel","year":"2023","journal-title":"J. Cyst. Fibros.ahead of print"},{"issue":"17","key":"10.1016\/j.pupt.2025.102345_bib30","doi-asserted-by":"crossref","first-page":"1612","DOI":"10.1056\/NEJMoa1807120","article-title":"VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles","volume":"379","author":"Keating","year":"2018","journal-title":"N. Engl. J. Med."},{"issue":"4","key":"10.1016\/j.pupt.2025.102345_bib31","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1016\/S2213-2600(22)00434-9","article-title":"Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials","volume":"11","author":"Mayer-Hamblett","year":"2023","journal-title":"Lancet Respir. Med."},{"key":"10.1016\/j.pupt.2025.102345_bib32","doi-asserted-by":"crossref","DOI":"10.1513\/AnnalsATS.202404-366OC","article-title":"Impact of discontinuing both hypertonic saline and dornase alfa after elexacaftor\/tezacaftor\/ivacaftor in cystic fibrosis","author":"Mayer-Hamblett","year":"2024","journal-title":"Ann Am Thorac Soc"},{"issue":"3","key":"10.1016\/j.pupt.2025.102345_bib33","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1016\/j.jcf.2024.02.003","article-title":"The effect of discontinuing hypertonic saline or dornase alfa on mucociliary clearance in elexacaftor\/tezacaftor\/ivacaftor treated people with cystic fibrosis: the SIMPLIFY-MCC Study","volume":"23","author":"Donaldson","year":"2024","journal-title":"J. Cyst. Fibros."}],"container-title":["Pulmonary Pharmacology & Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1094553925000021?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1094553925000021?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T04:08:29Z","timestamp":1777608509000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1094553925000021"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,3]]},"references-count":32,"alternative-id":["S1094553925000021"],"URL":"https:\/\/doi.org\/10.1016\/j.pupt.2025.102345","relation":{},"ISSN":["1094-5539"],"issn-type":[{"value":"1094-5539","type":"print"}],"subject":[],"published":{"date-parts":[[2025,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis","name":"articletitle","label":"Article Title"},{"value":"Pulmonary Pharmacology & Therapeutics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.pupt.2025.102345","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Ltd.","name":"copyright","label":"Copyright"}],"article-number":"102345"}}