{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T06:20:37Z","timestamp":1792131637599,"version":"4.4.0"},"reference-count":68,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2025,6,5]],"date-time":"2025-06-05T00:00:00Z","timestamp":1749081600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Fibrosis transmembrane conductance regulator (CFTR) modulators (CFTRms) have significantly improved outcomes in people with cystic fibrosis (CF). Real-world evidence, particularly from national and international CF registries, is essential to assess their long-term effectiveness and safety. We reviewed published studies using registry data to evaluate the impact of CFTRms on clinical outcomes in individuals with CF. A narrative review of studies published between 2015 and 2025 was conducted, focusing on registry-based evaluations of ivacaftor, lumacaftor\/ivacaftor, tezacaftor\/ivacaftor, and elexacaftor\/tezacaftor\/ivacaftor. Primary outcomes included lung function, pulmonary exacerbations, nutritional status, and survival. Fifty-seven registry-based studies confirmed the benefits of CFTRms across diverse CF populations. Ivacaftor has demonstrated sustained improvements in forced expiratory volume in one second (FEV1), reduced exacerbations, and improved nutritional outcomes. Lumacaftor\/ivacaftor and tezacaftor\/ivacaftor have shown modest benefits, especially in homozygous F508del patients. The introduction of elexacaftor\/tezacaftor\/ivacaftor has led to unprecedented improvements in lung function and quality of life, along with a reduced need for lung transplantation. Methodological heterogeneity and incomplete data remain challenges. Registry data provide essential, complementary evidence to clinical trials and support the effectiveness of CFTRms in routine care. Continued efforts are needed to harmonize registry methodologies and outcome measures.<\/jats:p>","DOI":"10.3390\/jcm14113978","type":"journal-article","created":{"date-parts":[[2025,6,5]],"date-time":"2025-06-05T03:58:52Z","timestamp":1749095932000},"page":"3978","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis: A Review of Registry-Based Evidence"],"prefix":"10.3390","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2432-415X","authenticated-orcid":false,"given":"Donatello","family":"Salvatore","sequence":"first","affiliation":[{"name":"Cystic Fibrosis Center, AOR Ospedale San Carlo, 85100 Potenza, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5801-0397","authenticated-orcid":false,"given":"Angela","family":"Pepe","sequence":"additional","affiliation":[{"name":"Cystic Fibrosis Center, AOR Ospedale San Carlo, 85100 Potenza, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,6,5]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1693","DOI":"10.1056\/NEJMra2216474","article-title":"Cystic Fibrosis","volume":"389","author":"Grasemann","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1361","DOI":"10.1164\/rccm.202202-0392OC","article-title":"Efficacy and Safety of Elexacaftor\/Tezacaftor\/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for","volume":"206","author":"Mall","year":"2022","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1016\/j.jcf.2017.11.013","article-title":"Epidemiology of CF: How registries can be used to advance our understanding of the CF population","volume":"17","author":"Jackson","year":"2018","journal-title":"J. Cyst. Fibros."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.jcf.2017.07.001","article-title":"Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting","volume":"17","author":"Hubert","year":"2018","journal-title":"J. Cyst. Fibros."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1007\/s41030-024-00269-9","article-title":"A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis","volume":"10","author":"Merlo","year":"2024","journal-title":"Pulm. Ther."},{"key":"ref_6","doi-asserted-by":"crossref","unstructured":"Szczesniak, R.D., Andrinopoulou, E.R., Li, H., Jain, R., Mayer-Hamblett, N., Ostrenga, J., Palipana, A.K., Pasta, D.J., Rosenfeld, M., and Todd, J. Real-world association between ivacaftor initiation and lung function variability: A registry study. J. Cyst. Fibros., 2025. in press.","DOI":"10.1016\/j.jcf.2025.01.014"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1016\/j.jcf.2020.06.012","article-title":"Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function","volume":"20","author":"Burgel","year":"2021","journal-title":"J. Cyst. Fibros."},{"key":"ref_8","first-page":"2101344","article-title":"Using chest computed tomography and unsupervised machine learning for predicting and evaluating response to lumacaftor-ivacaftor in people with cystic fibrosis","volume":"59","author":"Campredon","year":"2022","journal-title":"Eur. Respir. J."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/j.jcf.2021.06.002","article-title":"Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis","volume":"21","author":"Reix","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"e27473","DOI":"10.1002\/ppul.27473","article-title":"Introduction of Ivacaftor\/Lumacaftor in Children With Cystic Fibrosis Homozygous for F508del in the Netherlands: A Nationwide Real-Life Study","volume":"60","author":"Zwitserloot","year":"2025","journal-title":"Pediatr. Pulmonol."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1016\/j.jcf.2022.01.012","article-title":"Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis","volume":"21","author":"Martin","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"ref_12","first-page":"00339","article-title":"Lung function improvement on triple modulators: High-resolution, nationwide data from the Danish Cystic Fibrosis Cohort","volume":"10","author":"Qvist","year":"2024","journal-title":"ERJ Open Res."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"2401146","DOI":"10.1183\/13993003.01146-2024","article-title":"Impact of the expanded label for elexacaftor\/tezacaftor\/ivacaftor in people with cystic fibrosis with no F508del variant in the USA","volume":"64","author":"Cromwell","year":"2024","journal-title":"Eur. Respir. J."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"1080","DOI":"10.1016\/j.jcf.2024.04.010","article-title":"Changes in exercise capacity in people with Cystic Fibrosis after one year of Elexacaftor\/Tezacaftor\/Ivacaftor treatment\u2014A Danish prospective cohort","volume":"23","author":"Philipsen","year":"2024","journal-title":"J. Cyst. Fibros."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"1375","DOI":"10.1513\/AnnalsATS.201902-122OC","article-title":"Ivacaftor Is Associated with Reduced Lung Infection by Key Cystic Fibrosis Pathogens. A Cohort Study Using National Registry Data","volume":"16","author":"Frost","year":"2019","journal-title":"Ann. Am. Thorac. Soc."},{"key":"ref_16","doi-asserted-by":"crossref","unstructured":"Chesshyre, E.L.D., Enderby, B., Shore, A.C., Warren, F.C., and Warris, A. (2025). Longitudinal Study on Clinical Predictors for Allergic Bronchopulmonary Aspergillosis in Children and Young People with Cystic Fibrosis Highlights the Impact of Infection with Aspergillus and Pseudomonas and Ivacaftor Treatment. J. Fungi, 11.","DOI":"10.3390\/jof11020116"},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1016\/j.ijid.2023.11.013","article-title":"Registry. Use of elexacaftor\/tezacaftor\/ivacaftor leads to changes in detection frequencies of Staphylococcus aureus and Pseudomonas aeruginosa dependent on age and lung function in people with cystic fibrosis","volume":"139","author":"Dittrich","year":"2024","journal-title":"Int. J. Infect. Dis."},{"key":"ref_18","doi-asserted-by":"crossref","unstructured":"Pollak, M., Gambazza, S., Orenti, A., De Rose, V., Prais, D., Kerem, E., and Zahav, M.M. (2025). ECFSPR Steering Group. Respiratory infections after elexacaftor\/tezacaftor\/ivacaftor treatment in people with cystic fibrosis: Analysis of the European Cystic Fibrosis Society Patient Registry. ERJ Open Res., in press.","DOI":"10.1183\/23120541.01248-2024"},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1016\/j.jcf.2024.09.017","article-title":"Changes in factors associated with inhaled antibiotic prescriptions for people with cystic fibrosis over time in the U.S","volume":"24","author":"Muhlebach","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1016\/j.jcf.2024.01.011","article-title":"Pancreatic enzyme prescription following ivacaftor licensing: A retrospective analysis of the US and UK cystic fibrosis registries","volume":"23","author":"Calthorpe","year":"2024","journal-title":"J. Cyst. Fibros."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"1002","DOI":"10.1016\/j.jcf.2023.06.010","article-title":"Body mass index and nutritional intake following Elexacaftor\/Tezacaftor\/Ivacaftor modulator therapy in adults with cystic fibrosis","volume":"22","author":"Caley","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1016\/j.jcf.2022.03.010","article-title":"Obesity in Cystic fibrosis: Prevalence, trends and associated factors data from the US cystic fibrosis foundation patient registry","volume":"21","author":"Szentpetery","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1016\/j.jcf.2023.10.012","article-title":"Factors associated with pubertal growth outcomes in cystic fibrosis: Early Growth and Puberty in CF","volume":"23","author":"Patil","year":"2024","journal-title":"J. Cyst. Fibros."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/j.jcf.2023.11.005","article-title":"Decline in HbA1c during the first year of elexacaftor\/tezacaftor\/ivacaftor treatment in the Danish cystic fibrosis cohort: Short title: Decline in HbA1c after elexacaftor\/tezacaftor\/ivacaftor treatment","volume":"23","author":"Nielsen","year":"2024","journal-title":"J. Cyst. Fibros."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1513\/AnnalsATS.201807-449OC","article-title":"CFTRm Use Is Associated with Higher Hemoglobin Levels in Individuals with Cystic Fibrosis","volume":"16","author":"Gifford","year":"2019","journal-title":"Ann. Am. Thorac. Soc."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1513\/AnnalsATS.201802-149OC","article-title":"Longitudinal Trends in Real-World Outcomes after Initiation of Ivacaftor. A Cohort Study from the Cystic Fibrosis Registry of Ireland","volume":"16","author":"Kirwan","year":"2019","journal-title":"Ann. Am. Thorac. Soc."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1016\/j.jcf.2021.08.014","article-title":"Treatment patterns in people with cystic fibrosis: Have they changed since the introduction of ivacaftor?","volume":"21","author":"Granger","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1136\/thoraxjnl-2020-216265","article-title":"Projecting the impact of triple CFTRm therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials","volume":"77","author":"Keogh","year":"2022","journal-title":"Thorax"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1007\/s41030-025-00287-1","article-title":"Elexacaftor\/Tezacaftor\/Ivacaftor for Cystic Fibrosis: Impact on Hospitalizations and Health Care Resource Utilization in a Universal Health Care Setting","volume":"11","author":"Ankarfeldt","year":"2025","journal-title":"Pulm. Ther."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1016\/j.jcf.2024.11.004","article-title":"Impact of elexacaftor\/tezacaftor\/ivacaftor on utilization of routine therapies in cystic fibrosis: Danish nationwide register study","volume":"24","author":"Jensen","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1016\/j.jcf.2017.01.008","article-title":"Pregnancy among cystic fibrosis women in the era of CFTRms","volume":"16","author":"Heltshe","year":"2017","journal-title":"J. Cyst. Fibros."},{"key":"ref_32","first-page":"100829","article-title":"Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease","volume":"80","author":"Martin","year":"2021","journal-title":"Respir. Med. Res."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1164\/rccm.202308-1525OC","article-title":"Elexacaftor\/Tezacaftor\/Ivacaftor Treatment and Depression-related Events","volume":"209","author":"Ramsey","year":"2024","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_34","doi-asserted-by":"crossref","unstructured":"McGarry, L.J., Bhaiwala, Z., Lopez, A., Chandler, C., Pelligra, C.G., Rubin, J.L., and Liou, T.G. (2023). Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data. PLoS ONE, 18.","DOI":"10.1371\/journal.pone.0283479"},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"742","DOI":"10.1016\/j.jcf.2018.08.007","article-title":"Rate and predictors of prescription of lumacaftor\u2014Ivacaftor in the 18 months following approval in the United States","volume":"17","author":"Sawicki","year":"2018","journal-title":"J. Cyst. Fibros."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"1753466618820186","DOI":"10.1177\/1753466618820186","article-title":"Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor\/ivacaftor","volume":"13","author":"Rubin","year":"2019","journal-title":"Ther. Adv. Respir. Dis."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"46","DOI":"10.24110\/0031-403X-2024-103-5-46-56","article-title":"Efficacy and safety of the CFTR-modulator lumacaftor\/ivacaftor use in patients with cystic fibrosis in different age groups","volume":"103","author":"Kondratyeva","year":"2024","journal-title":"Pediatr. J. Named After G.N. Speransky"},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"897","DOI":"10.1080\/13696998.2024.2373000","article-title":"Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTRm, lumacaftor\/ivacaftor: An assessment using linked registry data in Sweden","volume":"27","author":"Lindblad","year":"2024","journal-title":"J. Med. Econ."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1016\/j.jcf.2020.07.017","article-title":"Projecting the impact of delayed access to elexacaftor\/tezacaftor\/ivacaftor for people with Cystic Fibrosis","volume":"20","author":"Stanojevic","year":"2021","journal-title":"J. Cyst. Fibros."},{"key":"ref_40","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."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"S92","DOI":"10.1016\/j.jval.2023.03.483","article-title":"CFTR-Modulators (CFTRM) Impact on Real-World Healthcare Resource Utilisation (HCRU) in Cystic Fibrosis (CF) in Wales: Retrospective, Observational, Database Study Using Linked Data from the UK CF Registry (UKCFR) and Sail-Databank","volume":"26","author":"Duran","year":"2023","journal-title":"Value Health"},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1016\/j.jcf.2024.10.006","article-title":"Factors associated with prescription of elexacaftor\/tezacaftor\/ivacaftor among people with cystic fibrosis aged 12 years or older with at least one F508del allele","volume":"24","author":"Hergenroeder","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"2302","DOI":"10.1002\/ppul.24854","article-title":"Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey","volume":"55","author":"Pekcan","year":"2020","journal-title":"Pediatr. Pulmonol."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.jcf.2024.07.015","article-title":"Characteristics of individuals with cystic fibrosis in the United States ineligible for ivacaftor and elexacaftor\/tezacaftor\/ivacaftor","volume":"24","author":"Sanders","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.jcf.2022.01.009","article-title":"Worldwide rates of diagnosis and effective treatment for cystic fibrosis","volume":"21","author":"Guo","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1007\/s41030-024-00274-y","article-title":"Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTRms","volume":"10","author":"Tomlinson","year":"2024","journal-title":"Pulm. Ther."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"2301241","DOI":"10.1183\/13993003.01241-2023","article-title":"Cystic fibrosis in Europe: Improved lung function and longevity\u2014Reasons for cautious optimism, but challenges remain","volume":"63","author":"Kerem","year":"2024","journal-title":"Eur. Respir. J."},{"key":"ref_48","doi-asserted-by":"crossref","unstructured":"Kondratyeva, E., Melyanovskaya, Y., Sherman, V., Voronkova, A., Zhekaite, E., Krasovsky, S., Amelina, E., Kashirskaya, N., Shadrina, V., and Polyakov, A. (2024). Study of the genetic and molecular epidemiology of cystic fibrosis based on the patient registry for planning targeted therapy in Russian Federation. Front. Genet., 15.","DOI":"10.3389\/fgene.2024.1383033"},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"22","DOI":"10.24953\/turkjpediatr.2025.4680","article-title":"Eligibility of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapies: Cohort of cystic fibrosis registry of T\u00fcrkiye","volume":"67","author":"Serbes","year":"2025","journal-title":"Turk. J. Pediatr."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1164\/rccm.201503-0578OC","article-title":"Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data","volume":"192","author":"Sawicki","year":"2015","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1136\/thoraxjnl-2017-210394","article-title":"Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor","volume":"73","author":"Bessonova","year":"2018","journal-title":"Thorax"},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.jcf.2019.05.015","article-title":"Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries","volume":"19","author":"Volkova","year":"2020","journal-title":"J. Cyst. Fibros."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1007\/s41030-020-00115-8","article-title":"Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012\u20132016 Experience","volume":"6","author":"Higgins","year":"2020","journal-title":"Pulm. Ther."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"1040","DOI":"10.1016\/j.jcf.2021.03.008","article-title":"Real-world use of ivacaftor in Canada: A retrospective analysis using the Canadian Cystic Fibrosis Registry","volume":"20","author":"Kawala","year":"2021","journal-title":"J. Cyst. Fibros."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1002\/ppul.25179","article-title":"Cystic fibrosis with non-G551D gating mutations in Italy: Epidemiology and clinical characteristics","volume":"56","author":"Salvatore","year":"2021","journal-title":"Pediatr. Pulmonol."},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1016\/j.jcf.2024.02.004","article-title":"Effectiveness of lumacaftor\/ivacaftor initiation in children with cystic fibrosis aged 2 through 5 years on disease progression: Interim results from an ongoing registry-based study","volume":"23","author":"Kim","year":"2024","journal-title":"J. Cyst. Fibros."},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1164\/rccm.201906-1227OC","article-title":"Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis","volume":"201","author":"Burgel","year":"2020","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1164\/rccm.202011-4153OC","article-title":"Rapid Improvement after Starting Elexacaftor-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease","volume":"204","author":"Burgel","year":"2021","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"730","DOI":"10.1016\/j.jcf.2023.03.002","article-title":"Real-world safety and effectiveness of elexacaftor\/tezacaftor\/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study","volume":"22","author":"Bower","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"189","DOI":"10.18093\/0869-0189-2023-33-2-189-197","article-title":"Efficacy of CFTRms in clinical practice (6-month follow-up)","volume":"33","author":"Kondratyeva","year":"2023","journal-title":"Pulmonologiya"},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"714","DOI":"10.1016\/j.jcf.2016.10.009","article-title":"Biomarkers for cystic fibrosis drug development","volume":"15","author":"Muhlebach","year":"2016","journal-title":"J. Cyst. Fibros."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/j.cca.2020.05.015","article-title":"Laboratory biomarkers for lung disease severity and progression in cystic fibrosis","volume":"508","author":"Bene","year":"2020","journal-title":"Clin. Chim. Acta"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1186\/s12931-023-02497-0","article-title":"The clinical impact of lumacaftor-ivacaftor on structural lung disease and lung function in children aged 6\u201311 with cystic fibrosis in a real-world setting","volume":"24","author":"McNally","year":"2023","journal-title":"Respir. Res."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1016\/j.jcf.2019.12.006","article-title":"Lumacaftor\/ivacaftor reduces exacerbations in adults homozygous for Phe508del mutation with severe lung disease","volume":"19","author":"Tong","year":"2020","journal-title":"J. Cyst. Fibros."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"75","DOI":"10.1513\/AnnalsATS.202002-144OC","article-title":"Clinical Effectiveness of Lumacaftor\/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial","volume":"18","author":"Sagel","year":"2021","journal-title":"Ann. Am. Thorac. Soc."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"381","DOI":"10.1164\/rccm.202008-3176LE","article-title":"Safety and efficacy of elexacaftor\/tezacaftor\/ivacaftor for 24 weeks or longer in people with cystic fibrosis and one or more F508del alleles: Interim results of an open-label phase 3 clinical trial","volume":"203","author":"Griese","year":"2020","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"ref_67","doi-asserted-by":"crossref","unstructured":"Daines, C.L., Polineni, D., Tullis, E., Costa, S., Linnemann, R.W., Mall, M.A., McKone, E.F., Quon, B.S., Ringshausen, F.C., and Selvadurai, H. Long-Term Safety and Efficacy of Elexacaftor\/Tezacaftor\/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Extension Study. Am. J. Respir. Crit. Care Med., 2025. in press.","DOI":"10.1164\/rccm.202411-2231OC"},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1016\/j.jcf.2018.03.001","article-title":"Cystic fibrosis patient registries: A valuable source for clinical research","volume":"17","author":"Dasenbrook","year":"2018","journal-title":"J. Cyst. Fibros."}],"container-title":["Journal of Clinical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2077-0383\/14\/11\/3978\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,9]],"date-time":"2025-10-09T17:46:52Z","timestamp":1760032012000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2077-0383\/14\/11\/3978"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,6,5]]},"references-count":68,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2025,6]]}},"alternative-id":["jcm14113978"],"URL":"https:\/\/doi.org\/10.3390\/jcm14113978","relation":{},"ISSN":["2077-0383"],"issn-type":[{"value":"2077-0383","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,6,5]]}}}