Multiple Breath Washout Is Feasible in the Clinical Setting and Detects Abnormal Lung Function in Infants and Young Children with Cystic Fibrosis

Stahl, Mirjam; Joachim, Cornelia; Blessing, Kerstin; Hämmerling, Susanne; Sommerburg, Olaf; Latzin, Philipp; Mall, Marcus

doi:10.1159/000357075

Cited by 54 publications

(54 citation statements)

References 33 publications

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“…These results strongly suggest that early rescue of CFTR function may be essential to prevent and/or delay irreversible lung damage in CF. As sensitive noninvasive outcome measures for assessment of abnormal lung structure and function in infants and young children, such as computed tomography, magnetic resonance imaging and multiple breath washout, are becoming available [28,38,[83][84][85][86], the ivacaftor clinical trial programme should be extended to studies in CF infants as soon as possible to exploit the narrow window of opportunity that has been created by widespread implementation of newborn screening and determine to what extent early therapy can delay or even prevent irreversible lung damage in CF.…”

Section: Can Cftr Modulator Therapy Prevent Cf Lung Disease?mentioning

confidence: 99%

CFTR: cystic fibrosis and beyond

2014

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Cystic fibrosis (CF) remains the most common fatal hereditary lung disease. The discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the stage for: 1) unravelling the molecular and cellular basis of CF lung disease; 2) the generation of animal models to study in vivo pathogenesis; and 3) the development of mutation-specific therapies that are now becoming available for a subgroup of patients with CF. This article highlights major advances in our understanding of how CFTR dysfunction causes chronic mucus obstruction, neutrophilic inflammation and bacterial infection in CF airways. Furthermore, we focus on recent breakthroughs and remaining challenges of novel therapies targeting the basic CF defect, and discuss the next steps to be taken to make disease-modifying therapies available to a larger group of patients with CF, including those carrying the most common mutation DF508-CFTR. Finally, we will summarise emerging evidence indicating that acquired CFTR dysfunction may be implicated in the pathogenesis of chronic obstructive pulmonary disease, suggesting that lessons learned from CF may be applicable to common airway diseases associated with mucus plugging. @ERSpublications CFTR dysfunction causes CF and may be implicated in more common chronic obstructive lung diseases, such as COPD

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Section: Can Cftr Modulator Therapy Prevent Cf Lung Disease?mentioning

confidence: 99%

CFTR: cystic fibrosis and beyond

2014

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“…In addition, it is feasible to perform the test in all age groups [7,[17][18][19], and there are now commercial devices available to facilitate testing in the clinical setting. Currently, less is known about the ability of LCI to detect treatment response in patients with moderate-to-severe lung disease; a population that is more likely to require hospitalisation for pulmonary exacerbations.…”

Section: Introductionmentioning

confidence: 99%

Lung clearance index in cystic fibrosis subjects treated for pulmonary exacerbations

et al. 2015

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Pulmonary exacerbations are important clinical events for cystic fibrosis (CF) patients. Studies assessing the ability of the lung clearance index (LCI) to detect treatment response for pulmonary exacerbations have yielded heterogeneous results. Here, we conduct a retrospective analysis of pooled LCI data to assess treatment with intravenous antibiotics for pulmonary exacerbations and to understand factors explaining the heterogeneous response.A systematic literature search was performed to identify prospective observational studies. Factors predicting the relative change in LCI and spirometry were evaluated while adjusting for within-study clustering.Six previously reported studies and one unpublished study, which included 176 pulmonary exacerbations in both paediatric and adult patients, were included. Overall, LCI significantly decreased by 0.40 units (95% CI −0.60-−0.19, p=0.004) or 2.5% following treatment. The relative change in LCI was significantly correlated with the relative change in forced expiratory volume in 1 s (FEV1), but results were discordant in 42.5% of subjects (80 out of 188). Higher (worse) baseline LCI was associated with a greater improvement in LCI (slope: −0.9%, 95% CI −1.0-−0.4%).LCI response to therapy for pulmonary exacerbations is heterogeneous in CF patients; the overall effect size is small and results are often discordant with FEV1. @ERSpublications Lung clearance index response to therapy for pulmonary exacerbations is heterogeneous in cystic fibrosis patients http://ow.ly/Mnvtd

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“…In contrast, STAHL et al [48] found a significantly higher LCI in CF children with a mean age of 1.3¡1.0 years compared with controls, implicating the importance of screening and early treatment to keep the lungs normal.…”

Section: Cystic Fibrosismentioning

confidence: 84%