Leveraging early markers of cystic fibrosis structural lung disease to improve outcomes

Flume, Patrick A.; VanDevanter, Donald R.

doi:10.1183/13993003.00105-2020

Cited by 1 publication

(2 citation statements)

References 13 publications

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“…The prevention of early lung disease is a priority for researchers, clinicians, CF patients and their families 30 . We currently possess tools and expertise that may be used to assess early lung disease progression.…”

Section: Discussionmentioning

confidence: 99%

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Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis

Bayfield

Douglas

Rosenow

et al. 2021

Thorax

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Structural and functional defects within the lungs of children with cystic fibrosis (CF) are detectable soon after birth and progress throughout preschool years often without overt clinical signs or symptoms. By school age, most children have structural changes such as bronchiectasis or gas trapping/hypoperfusion and lung function abnormalities that persist into later life. Despite improved survival, gains in forced expiratory volume in one second (FEV1) achieved across successive birth cohorts during childhood have plateaued, and rates of FEV1 decline in adolescence and adulthood have not slowed. This suggests that interventions aimed at preventing lung disease should be targeted to mild disease and commence in early life. Spirometry-based classifications of ‘normal’ (FEV1≥90% predicted) and ‘mild lung disease’ (FEV1 70%–89% predicted) are inappropriate, given the failure of spirometry to detect significant structural or functional abnormalities shown by more sensitive imaging and lung function techniques. The state and readiness of two imaging (CT and MRI) and two functional (multiple breath washout and oscillometry) tools for the detection and monitoring of early lung disease in children and adults with CF are discussed in this article.Prospective research programmes and technological advances in these techniques mean that well-designed interventional trials in early lung disease, particularly in young children and infants, are possible. Age appropriate, randomised controlled trials are critical to determine the safety, efficacy and best use of new therapies in young children. Regulatory bodies continue to approve medications in young children based on safety data alone and extrapolation of efficacy results from older age groups. Harnessing the complementary information from structural and functional tools, with measures of inflammation and infection, will significantly advance our understanding of early CF lung disease pathophysiology and responses to therapy. Defining clinical utility for these novel techniques will require effective collaboration across multiple disciplines to address important remaining research questions. Future impact on existing management burden for patients with CF and their family must be considered, assessed and minimised.To address the possible role of these techniques in early lung disease, a meeting of international leaders and experts in the field was convened in August 2019 at the Australiasian Cystic Fibrosis Conference. The meeting entitiled ‘Shaping imaging and functional testing for early disease detection of lung disease in Cystic Fibrosis’, was attended by representatives across the range of disciplines involved in modern CF care. This document summarises the proceedings, key priorities and important research questions highlighted.

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Section: Discussionmentioning

confidence: 99%

“…Despite the universal use of spirometry, the minimum clinically important difference (MCID) remains unknown. A revised definition based on sensitive surrogates of early lung disease is needed to expedite the use of novel techniques as end-points in future intervention studies 30 .…”

Section: Current Status Of Lung Function Techniquesmentioning

confidence: 99%

Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis

Bayfield

Douglas

Rosenow

et al. 2021

Thorax

View full text Add to dashboard Cite

show abstract

Leveraging early markers of cystic fibrosis structural lung disease to improve outcomes

Abstract: Wijker and colleagues have demonstrated the link of inflammation and obstruction to eventual structural disease in CF. Now we may have another clinical endpoint by which we can judge the impact of early interventions.

Cited by 1 publication

References 13 publications

Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis

Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis

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