三重调节剂改善肺功能:来自丹麦囊性纤维化队列的高分辨率全国数据

IF 4.3 3区 医学 Q1 RESPIRATORY SYSTEM
ERJ Open Research Pub Date : 2024-12-09 eCollection Date: 2024-11-01 DOI:10.1183/23120541.00339-2024
Christian Leo-Hansen, Daniel Faurholt-Jepsen, Tavs Qvist, Christine Højte, Bibi U Nielsen, Thomas Bryrup, Esben H Henriksen, Terese Katzenstein, Marianne Skov, Inger H M Mathiesen, Majbritt Jeppesen, Søren Jensen-Fangel, Hanne V Olesen, Frederik Fouirnaies Buchvald, Kim Gjerum Nielsen, Espen Jimenez-Solem, Christian Ritz, Tacjana Pressler, Mette F Olsen
{"title":"三重调节剂改善肺功能:来自丹麦囊性纤维化队列的高分辨率全国数据","authors":"Christian Leo-Hansen, Daniel Faurholt-Jepsen, Tavs Qvist, Christine Højte, Bibi U Nielsen, Thomas Bryrup, Esben H Henriksen, Terese Katzenstein, Marianne Skov, Inger H M Mathiesen, Majbritt Jeppesen, Søren Jensen-Fangel, Hanne V Olesen, Frederik Fouirnaies Buchvald, Kim Gjerum Nielsen, Espen Jimenez-Solem, Christian Ritz, Tacjana Pressler, Mette F Olsen","doi":"10.1183/23120541.00339-2024","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>People living with cystic fibrosis in Denmark had early, universal access to triple modulator treatment with elexacaftor/tezacaftor/ivacaftor. Close monitoring allowed us to assess the impact of treatment on lung function and progression of lung disease in an unselected nationwide cystic fibrosis population from 6 years of age.</p><p><strong>Methods: </strong>Data were analysed using linear mixed-effect models to assess changes in levels and annual rates of change (slopes) in percent predicted (pp) forced expiratory volume in 1 s (FEV<sub>1</sub>), forced vital capacity (FVC) and forced expiratory flow at 25-75% of FVC (ppFEF<sub>25-75%</sub>) between the 12 months pre-treatment and treatment periods. Subgroup analyses assessed the impact of elexacaftor/tezacaftor/ivacaftor among those with/without previous modulator treatment, normal/mild/moderate/severe lung disease at treatment initiation, children/adults and birth cohorts.</p><p><strong>Results: </strong>We included 392 people living with cystic fibrosis with a median (interquartile range) 12 (nine to 15) spirometry measurements per person. The mean (95% CI) improvement in ppFEV<sub>1</sub> was 13.0 (11.3-14.6) 12 months after initiation of elexacaftor/tezacaftor/ivacaftor treatment. The annual rate of change improved from -1.4 (-2.1 - -0.6) ppFEV<sub>1</sub> in the pre-treatment year to 2.7 (1.8-3.5) ppFEV<sub>1</sub> per year during treatment. Similarly, ppFVC increased by 8.0 (7.1-8.9) and FEF<sub>25--75%</sub> by 19.5 (17.0-21.9).</p><p><strong>Conclusions: </strong>Using high-resolution data from a nationwide real-world setting, our study documents the impact of elexacaftor/tezacaftor/ivacaftor on lung function across subgroups based on age, disease severity and treatment history. These findings point towards a new period of consistent lung function improvement among people living with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor.</p>","PeriodicalId":11739,"journal":{"name":"ERJ Open Research","volume":"10 6","pages":""},"PeriodicalIF":4.3000,"publicationDate":"2024-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11626609/pdf/","citationCount":"0","resultStr":"{\"title\":\"Lung function improvement on triple modulators: high-resolution, nationwide data from the Danish Cystic Fibrosis Cohort.\",\"authors\":\"Christian Leo-Hansen, Daniel Faurholt-Jepsen, Tavs Qvist, Christine Højte, Bibi U Nielsen, Thomas Bryrup, Esben H Henriksen, Terese Katzenstein, Marianne Skov, Inger H M Mathiesen, Majbritt Jeppesen, Søren Jensen-Fangel, Hanne V Olesen, Frederik Fouirnaies Buchvald, Kim Gjerum Nielsen, Espen Jimenez-Solem, Christian Ritz, Tacjana Pressler, Mette F Olsen\",\"doi\":\"10.1183/23120541.00339-2024\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>People living with cystic fibrosis in Denmark had early, universal access to triple modulator treatment with elexacaftor/tezacaftor/ivacaftor. Close monitoring allowed us to assess the impact of treatment on lung function and progression of lung disease in an unselected nationwide cystic fibrosis population from 6 years of age.</p><p><strong>Methods: </strong>Data were analysed using linear mixed-effect models to assess changes in levels and annual rates of change (slopes) in percent predicted (pp) forced expiratory volume in 1 s (FEV<sub>1</sub>), forced vital capacity (FVC) and forced expiratory flow at 25-75% of FVC (ppFEF<sub>25-75%</sub>) between the 12 months pre-treatment and treatment periods. Subgroup analyses assessed the impact of elexacaftor/tezacaftor/ivacaftor among those with/without previous modulator treatment, normal/mild/moderate/severe lung disease at treatment initiation, children/adults and birth cohorts.</p><p><strong>Results: </strong>We included 392 people living with cystic fibrosis with a median (interquartile range) 12 (nine to 15) spirometry measurements per person. The mean (95% CI) improvement in ppFEV<sub>1</sub> was 13.0 (11.3-14.6) 12 months after initiation of elexacaftor/tezacaftor/ivacaftor treatment. The annual rate of change improved from -1.4 (-2.1 - -0.6) ppFEV<sub>1</sub> in the pre-treatment year to 2.7 (1.8-3.5) ppFEV<sub>1</sub> per year during treatment. Similarly, ppFVC increased by 8.0 (7.1-8.9) and FEF<sub>25--75%</sub> by 19.5 (17.0-21.9).</p><p><strong>Conclusions: </strong>Using high-resolution data from a nationwide real-world setting, our study documents the impact of elexacaftor/tezacaftor/ivacaftor on lung function across subgroups based on age, disease severity and treatment history. These findings point towards a new period of consistent lung function improvement among people living with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor.</p>\",\"PeriodicalId\":11739,\"journal\":{\"name\":\"ERJ Open Research\",\"volume\":\"10 6\",\"pages\":\"\"},\"PeriodicalIF\":4.3000,\"publicationDate\":\"2024-12-09\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11626609/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"ERJ Open Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1183/23120541.00339-2024\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/11/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"ERJ Open Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1183/23120541.00339-2024","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/11/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
引用次数: 0

摘要

背景:丹麦的囊性纤维化患者很早就普遍获得了elexaftor /tezacaftor/ivacaftor的三重调节剂治疗。密切监测使我们能够在未选择的全国性6岁囊性纤维化人群中评估治疗对肺功能和肺病进展的影响。方法:使用线性混合效应模型对数据进行分析,以评估治疗前和治疗期12个月期间预测(pp) 1秒内强制呼气量(FEV1)、强制肺活量(FVC)和FVC 25-75%的强制呼气流量(ppFEF25-75%)的水平和年变化率(斜率)的变化。亚组分析评估了elexaftor /tezacaftor/ivacaftor对既往接受过/未接受过调节剂治疗、治疗开始时患有正常/轻度/中度/重度肺部疾病、儿童/成人和出生队列的影响。结果:我们纳入了392例囊性纤维化患者,平均每人肺活量测量值为12(9至15)。开始elexaftor /tezacaftor/ivacaftor治疗后12个月ppFEV1的平均(95% CI)改善为13.0(11.3-14.6)。年变化率从治疗前一年的-1.4 (-2.1 - -0.6)ppFEV1改善到治疗期间的2.7 (1.8-3.5)ppFEV1。ppFVC增加了8.0 (7.1 ~ 8.9),FEF25—75%增加了19.5(17.0 ~ 21.9)。结论:我们的研究使用来自全国真实世界的高分辨率数据,记录了elexaftor /tezacaftor/ivacaftor对基于年龄、疾病严重程度和治疗史的亚组肺功能的影响。这些发现表明,囊性纤维化患者使用elexaftor /tezacaftor/ivacaftor后,肺功能将持续改善。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Lung function improvement on triple modulators: high-resolution, nationwide data from the Danish Cystic Fibrosis Cohort.

Background: People living with cystic fibrosis in Denmark had early, universal access to triple modulator treatment with elexacaftor/tezacaftor/ivacaftor. Close monitoring allowed us to assess the impact of treatment on lung function and progression of lung disease in an unselected nationwide cystic fibrosis population from 6 years of age.

Methods: Data were analysed using linear mixed-effect models to assess changes in levels and annual rates of change (slopes) in percent predicted (pp) forced expiratory volume in 1 s (FEV1), forced vital capacity (FVC) and forced expiratory flow at 25-75% of FVC (ppFEF25-75%) between the 12 months pre-treatment and treatment periods. Subgroup analyses assessed the impact of elexacaftor/tezacaftor/ivacaftor among those with/without previous modulator treatment, normal/mild/moderate/severe lung disease at treatment initiation, children/adults and birth cohorts.

Results: We included 392 people living with cystic fibrosis with a median (interquartile range) 12 (nine to 15) spirometry measurements per person. The mean (95% CI) improvement in ppFEV1 was 13.0 (11.3-14.6) 12 months after initiation of elexacaftor/tezacaftor/ivacaftor treatment. The annual rate of change improved from -1.4 (-2.1 - -0.6) ppFEV1 in the pre-treatment year to 2.7 (1.8-3.5) ppFEV1 per year during treatment. Similarly, ppFVC increased by 8.0 (7.1-8.9) and FEF25--75% by 19.5 (17.0-21.9).

Conclusions: Using high-resolution data from a nationwide real-world setting, our study documents the impact of elexacaftor/tezacaftor/ivacaftor on lung function across subgroups based on age, disease severity and treatment history. These findings point towards a new period of consistent lung function improvement among people living with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
ERJ Open Research
ERJ Open Research Medicine-Pulmonary and Respiratory Medicine
CiteScore
6.20
自引率
4.30%
发文量
273
审稿时长
8 weeks
期刊介绍: ERJ Open Research is a fully open access original research journal, published online by the European Respiratory Society. The journal aims to publish high-quality work in all fields of respiratory science and medicine, covering basic science, clinical translational science and clinical medicine. The journal was created to help fulfil the ERS objective to disseminate scientific and educational material to its members and to the medical community, but also to provide researchers with an affordable open access specialty journal in which to publish their work.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信