卢美他尼/依伐卡托对囊性纤维化患者呼吸道细菌和真菌病原体的影响：瑞典一项前瞻性多中心队列研究。

Impact of lumacaftor/ivacaftor on the bacterial and fungal respiratory pathogens in cystic fibrosis: a prospective multicenter cohort study in Sweden.

机构信息

Stockholm CF Centre, Karolinska University Hospital Huddinge, Stockholm, Department of Clinical Science, Intervention and Technology, Sweden.

Division of Pediatrics, Karolinska Institutet, Alfred Nobels Allé 8, Stockholm 171 77, Sweden.

出版信息

Ther Adv Respir Dis. 2024 Jan-Dec;18:17534666241254090. doi: 10.1177/17534666241254090.

BACKGROUND

A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.

OBJECTIVES

The aim of this study was to determine the impact of lumacaftor/ivacaftor on the bacterial and fungal burden.

DESIGN

The study is a prospective multicenter cohort study including 132 CF patients homozygous for F508del treated with lumacaftor/ivacaftor.

METHODS

Clinical parameters as well as bacterial and fungal outcomes 1 year after initiation of lumacaftor/ivacaftor were compared to data from 2 years prior to initiation of the treatment. Changes in the slope of the outcomes before and after the onset of treatment were assessed.

RESULTS

Lung function measured as ppFEV1 ( < 0.001), body mass index (BMI) in adults ( < 0.001), and BMI -score in children ( = 0.007) were improved after initiation of lumacaftor/ivacaftor. In addition, the slope of the prevalence of ( = 0.007) and ( < 0.001) shifted from positive to negative, that is, became less prevalent, 1 year after treatment, while the slope for ( = 0.009), spp ( = 0.026), and ( < 0.001) shifted from negative to positive.

CONCLUSION

The current study showed a significant improvement in clinical parameters and a reduction of some of CF respiratory microorganisms 1 year after starting with lumacaftor/ivacaftor. However, no significant changes were observed for , or , key pathogens in the CF context.

背景

在接受 lumacaftor/ivacaftor 治疗的 F508del 纯合子 CF 患者中，肺部恶化率显著下降。然而，目前尚不清楚这种减少是否反映了微生物负担的减轻。

目的

本研究旨在确定 lumacaftor/ivacaftor 对细菌和真菌负担的影响。

设计

这是一项前瞻性多中心队列研究，包括 132 名接受 lumacaftor/ivacaftor 治疗的 F508del 纯合子 CF 患者。

方法

比较 lumacaftor/ivacaftor 治疗开始前 1 年与治疗开始前 2 年的临床参数以及细菌和真菌结果。评估治疗前后结果斜率的变化。

结果

肺功能（ppFEV1）（<0.001）、成人 BMI（<0.001）和儿童 BMI 评分（=0.007）在开始使用 lumacaftor/ivacaftor 后得到改善。此外，（=0.007）和（<0.001）的流行率斜率从正变为负，即在治疗 1 年后变得不那么流行，而（=0.009）、 spp（=0.026）和（<0.001）的斜率从负变为正。