Nationwide lung function monitoring from infancy in newborn-screened children with cystic fibrosis

Author:

Sandvik Rikke M.,Schmidt Marika N.,Voldby Christian M.ORCID,Buchvald Frederik F.,Olesen Hanne V.,Olsen Jørgen,Kragh Maja V.,Rubak Sune L.M.,Pressler Tacjana,Robinson Paul D.ORCID,Gustafsson Per M.,Skov Marianne,Nielsen Kim G.

Abstract

BackgroundCystic fibrosis (CF) lung disease starts in infancy and can be assessed for structural lung abnormalities using computed tomography or magnetic resonance scans, or for lung function impairment using multiple breath washout (MBW). However, in infancy these two methods are not well correlated. Trajectories of CF lung disease assessed by MBW in infants and toddlers remain poorly described, which is why we aimed to 1) describe the trajectory of lung function, 2) explore risk factors for progression and 3) explore the real-life effect of lumacaftor/ivacaftor.MethodsThis was a nationwide observational cohort study (2018–2021) using data collected as part of the routine clinical surveillance programme (including MBW and monthly endo-laryngeal suction sampling for bacterial pathogens) in children born after implementation of newborn screening for CF (May 2016). Lumacaftor/ivacaftor commenced from age 2 years in children homozygous for F508del. Ventilation distribution efficiency (VDE), recently described to have advantages over lung clearance index (LCI), was reported as the primary MBW outcome after z-score calculations based on published reference data. Mixed effect linear regression models were the main statistical analyses performed in this study.Results59 children, aged 2–45 months, contributed with 211 MBW occasions (median (interquartile range (IQR)) 3 (2–5) MBW occasions per child) with a median (IQR) follow-up time of 10.8 (5.2–22.3) months. An overall mean annual deterioration rate of −0.50 (95% CI −0.78– −0.22) z-VDE was observed, starting from an estimated mean z-VDE of −1.68 (95% CI −2.15– −1.22) at age 0.0 years (intercept).Pseudomonas aeruginosa“ever” (n=14, MBWs 50) had a significantly worse z-VDE trajectoryversus P. aeruginosa“never” (mean difference 0.53 (95% CI 0.16–0.89) per year; p=0.0047) and lumacaftor/ivacaftor treatment (n=22, MBWs 46) significantly improved the trajectory of z-VDE (mean difference 1.72 (95% CI 0.79–2.66) per year; p=0.0004), leading to a stable mean z-VDE trajectory after start of treatment.ConclusionsInfants and toddlers with CF demonstrated progressive deterioration in z-VDE over the first years of life.P. aeruginosaisolation “ever” was associated with an accelerated deterioration in lung function, while lumacaftor/ivacaftor therapy significantly improved and stabilised the trajectory.

Funder

Rigshospitalets research foundation

Børnelungefonden

Tømrermester Jørgen Holm og Hustru Elisa f. Hansens Mindelegat

Publisher

European Respiratory Society (ERS)

Subject

Pulmonary and Respiratory Medicine

Cited by 1 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3