LONGITUDE: An observational study of the long-term effectiveness of elexacaftor/tezacaftor/ivacaftor in people aged ≥12 years with cystic fibrosis using data from the United Kingdom Cystic Fibrosis Registry - 2-year analysis.
Gabriela Vega-Hernandez, Gordon MacGregor, Andrew Wilfin, Francis Adams, Ciarán Haugh, Carl A Baxter, Heike Wöhling, Sarah L Clarke, Susan C Charman, Siobhán B Carr
{"title":"LONGITUDE: An observational study of the long-term effectiveness of elexacaftor/tezacaftor/ivacaftor in people aged ≥12 years with cystic fibrosis using data from the United Kingdom Cystic Fibrosis Registry - 2-year analysis.","authors":"Gabriela Vega-Hernandez, Gordon MacGregor, Andrew Wilfin, Francis Adams, Ciarán Haugh, Carl A Baxter, Heike Wöhling, Sarah L Clarke, Susan C Charman, Siobhán B Carr","doi":"10.1016/j.jcf.2025.04.012","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>The cystic fibrosis (CF) transmembrane conductance regulator modulator (CFTRm) elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has demonstrated efficacy and safety in clinical trials and emerging observational studies in people with CF. This study evaluated the real-world impact of ELX/TEZ/IVA in a large cohort of people with CF in the UK.</p><p><strong>Methods: </strong>LONGITUDE is an observational, registry-based cohort study using data from the UK CF Registry to evaluate outcomes of ELX/TEZ/IVA in people aged ≥6 years who initiated ELX/TEZ/IVA from August 2019. Key outcomes included percent predicted forced expiratory volume in 1 s (ppFEV<sub>1</sub>), body mass index (BMI), pulmonary exacerbations (PEx), lung infections, transplants, deaths, and treatment discontinuation. We report results of people ≥12 years with data up to December 31, 2022.</p><p><strong>Results: </strong>A total of 5187 people were included (mean follow-up 19.1 months). ppFEV<sub>1</sub> improvements were observed at 2 years (10.2; 95 % CI: 9.6, 10.8; n = 1448). A clinically meaningful difference in the annual change of ppFEV<sub>1</sub> between ELX/TEZ/IVA-treated people and historical CFTRm-naïve controls was observed, with those treated with ELX/TEZ/IVA having less of a decline in lung function over time by 1.1 percentage points (95 % CI: 0.9, 1.4). A 64.7 % reduction in the rate of PEx, increase in BMI by 1.7 kg/m<sup>2</sup> (SD: 2.3), reduced lung infections, and low number of lung transplants and deaths were also observed.</p><p><strong>Conclusions: </strong>People with CF aged ≥12 years in the UK who initiated ELX/TEZ/IVA had sustained improvements in multiple CF-related health outcomes, consistent with results from clinical trials. These results support the positive impact of ELX/TEZ/IVA on the lives of people with CF.</p>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":" ","pages":""},"PeriodicalIF":5.4000,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Cystic Fibrosis","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jcf.2025.04.012","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
引用次数: 0
Abstract
Background: The cystic fibrosis (CF) transmembrane conductance regulator modulator (CFTRm) elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has demonstrated efficacy and safety in clinical trials and emerging observational studies in people with CF. This study evaluated the real-world impact of ELX/TEZ/IVA in a large cohort of people with CF in the UK.
Methods: LONGITUDE is an observational, registry-based cohort study using data from the UK CF Registry to evaluate outcomes of ELX/TEZ/IVA in people aged ≥6 years who initiated ELX/TEZ/IVA from August 2019. Key outcomes included percent predicted forced expiratory volume in 1 s (ppFEV1), body mass index (BMI), pulmonary exacerbations (PEx), lung infections, transplants, deaths, and treatment discontinuation. We report results of people ≥12 years with data up to December 31, 2022.
Results: A total of 5187 people were included (mean follow-up 19.1 months). ppFEV1 improvements were observed at 2 years (10.2; 95 % CI: 9.6, 10.8; n = 1448). A clinically meaningful difference in the annual change of ppFEV1 between ELX/TEZ/IVA-treated people and historical CFTRm-naïve controls was observed, with those treated with ELX/TEZ/IVA having less of a decline in lung function over time by 1.1 percentage points (95 % CI: 0.9, 1.4). A 64.7 % reduction in the rate of PEx, increase in BMI by 1.7 kg/m2 (SD: 2.3), reduced lung infections, and low number of lung transplants and deaths were also observed.
Conclusions: People with CF aged ≥12 years in the UK who initiated ELX/TEZ/IVA had sustained improvements in multiple CF-related health outcomes, consistent with results from clinical trials. These results support the positive impact of ELX/TEZ/IVA on the lives of people with CF.
期刊介绍：
The Journal of Cystic Fibrosis is the official journal of the European Cystic Fibrosis Society. The journal is devoted to promoting the research and treatment of cystic fibrosis. To this end the journal publishes original scientific articles, editorials, case reports, short communications and other information relevant to cystic fibrosis. The journal also publishes news and articles concerning the activities and policies of the ECFS as well as those of other societies related the ECFS.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
