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Progression of Lung Disease in Preschool Patients with Cystic Fibrosis.

Abstract：

RATIONALE:Implementation of intervention strategies to prevent lung damage in early cystic fibrosis (CF) requires objective outcome measures that capture and track lung disease. OBJECTIVES:To define the utility of the Lung Clearance Index (LCI), measured by multiple breath washout, as a means to track disease progression in preschool children with CF. METHODS:Children with CF between the ages of 2.5 and 6 years with a confirmed diagnosis of CF and age-matched healthy control subjects were enrolled at three North American CF centers. Multiple breath washout tests were performed at baseline, 1, 3, 6, and 12 months to mimic time points chosen in clinical care and interventional trials; spirometry was also conducted. A generalized linear mixed-effects model was used to distinguish LCI changes associated with normal growth and development (i.e., healthy children) from the progression of CF lung disease. MEASUREMENTS AND MAIN RESULTS:Data were collected on 156 participants with 800 LCI measurements. Although both LCI and spirometry discriminated health from disease, only the LCI identified significant deterioration of lung function in CF over time. The LCI worsened during cough episodes and pulmonary exacerbations, whereas similar symptoms in healthy children were not associated with increased LCI values. CONCLUSIONS:LCI is a useful marker to track early disease progression and may serve as a tool to guide therapies in young patients with CF.

journal_name

Am J Respir Crit Care Med

journal_title

American journal of respiratory and critical care medicine

authors

Stanojevic S,Davis SD,Retsch-Bogart G,Webster H,Davis M,Johnson RC,Jensen R,Pizarro ME,Kane M,Clem CC,Schornick L,Subbarao P,Ratjen FA

doi

10.1164/rccm.201610-2158OC

subject

Has Abstract

pub_date

2017-05-01 00:00:00

pages

1216-1225

issue

9

eissn

1073-449X

issn

1535-4970

journal_volume

195

pub_type

杂志文章,多中心研究

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