WS15.05Elexacaftor/tezacafor/ivacaftor improves bronchial dilatation in a real-world cohort of adolescents with cystic fibrosis

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis Pub Date : 2025-06-01 DOI:10.1016/j.jcf.2025.03.580

A.-S. Bonnel , I. Sermet-Gaudelus , A. Letierce , L. Berteloot , P. Makani , D. Caudri , H. Tiddens , Y. Chen , Modul-CF Study Group

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引用次数: 0

Abstract

Objective

Elexacaftor/ tezacaftor/ivacaftor (ETI) improves clinical status of adolescents with cystic fibrosis (aCF). The impact on lung structure is unclear in the pediatric population.

Methods

MODUl-CF is a real-world study of the French pediatric cohort treated by ETI over 5 years. Pulmonary Function Tests include Forced Expiratory Volume in 1 second (FEV₁), Forced Expiratory Flow at 25-75 (FEF_25-75), Residual Volume/Total Lung Capacity (RV/TLC) and, for a subset of patient, Lung Clearance Index and sputum inflammatory markers. Low dose inspiratory controlled chest CT images are assessed using the LungQ^TM platform (Thirona, Nijmegen) bronchus-artery (BA) analysis, automated PRAGMA-CF analysis, and the Mucus Plugging (MP) analysis. The BA-analysis measures the dimensions of all detectable bronchi and their adjacent arteries e.g; the bronchial outer diameter (B_out), the bronchial wall thickness (B_wt) all calculated relative to the adjacent artery diameter (A). A transformation back calculates the volume of bronchiectasis (%Bx), bronchial wall thickening (%Bwt), all expressed as percentage of total lung volume (TLV). Bronchiectasis are defined by B_out/A >1.5.

Results

All B/A ratios improved significantly in 188 patients who had a chest CT at baseline and M12. Markers of bronchial wall thickening, %Bwt, B_wtA decreased by -0.6 (-1.23; 0.08) (median(Q1; Q3); -0.08(-0.15; -0.01) (p<0.0001). Mucus plugs number and volume decreased substantially to a value approaching 0. Measures of bronchiectasis (%Bx and B_outA ratio) decreased significantly by -1.42 (-3.84; -0.1) and -0.09 (-0.2; -0.01); p< 0.0001. This correlated with improvement in bronchial pro-inflammatory parameters, RV/TLC and LCI. The number of BA pairs with B_out/A >1.5 decreased significantly from 40%(19) at M0 to 28%(17) at M12 (p<0.0001). This decrease was positively correlated with M0 and M12 B_out/A, B_wtA, mucus plug volume, ppRV/TLC and ppTLV (p<0.0001). The decrease at M12 in number of pairs > 1.5 was correlated with the variation of FEF_25-75 at M1 and M12.

Conclusions

ETI improves significantly bronchial dilatation and bronchial wall thickness in aCF. Our data suggest a decrease in the number of bronchiectasis at 1 year ETI in correlation with improvement in bronchial inflammation, lung distension and mucus distal bronchial obstruction.

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elexacaftor /tezacafor/ivacaftor可改善囊性纤维化青少年患者的支气管扩张

目的：改善青少年囊性纤维化（aCF）患者的临床状况。对儿科人群肺结构的影响尚不清楚。smodul - cf是一项对接受ETI治疗超过5年的法国儿童队列的现实研究。肺功能测试包括1秒用力呼气量（FEV1）、25-75用力呼气流量（FEF25-75）、残气量/总肺活量（RV/TLC），以及部分患者的肺清除率指数和痰炎标志物。使用LungQTM平台（Thirona, Nijmegen）支气管动脉（BA）分析、自动化PRAGMA-CF分析和黏液堵塞（MP）分析评估低剂量吸气控制胸部CT图像。ba分析测量所有可检测到的支气管及其邻近动脉的尺寸，例如；支气管外径（Bout）、支气管壁厚（Bwt）均相对于邻近动脉直径(A)计算。转换后计算支气管扩张的体积（%Bx），支气管壁增厚（%Bwt），均以总肺体积（TLV）的百分比表示。根据Bout/A >；1.5定义支气管扩张。结果188例基线及M12期胸部CT检查患者B/A比值均显著提高。支气管壁增厚指标，%Bwt， BwtA下降-0.6 (-1.23；0.08)(中值(Q1;第三季度);-0.08 (-0.15;-0.01)(术中,0.0001)。粘液塞的数量和体积大幅减少，接近于0。支气管扩张测量值（%Bx和BoutA比值）显著降低-1.42 (-3.84；-0.1)和-0.09 (-0.2；-0.01);术中;0.0001. 这与支气管促炎参数、RV/TLC和LCI的改善相关。Bout/A >；1.5的BA对数量从M0时的40%（19对）显著下降到M12时的28%（17对）（p<0.0001）。这种降低与M0和M12、Bout/A、BwtA、粘液塞体积、ppRV/TLC和ppTLV呈正相关（p<0.0001）。M12时对数的减少>；1.5与M1和M12时FEF25-75的变异相关。结论seti可明显改善aCF患者的支气管扩张和支气管壁厚度。我们的数据表明，1年ETI时支气管扩张的数量减少与支气管炎症、肺扩张和粘液远端支气管阻塞的改善相关。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Cystic Fibrosis 医学-呼吸系统

CiteScore

10.10

自引率

13.50%

发文量

1361

审稿时长

50 days

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.