PRAGMA-CF. A quantitative structural lung disease CT outcome in young children with cystic fibrosis

Tim Rosenow, Merel C.J. Oudraad, Conor P. Murray, Lidija Turkovic, Wieying Kuo, Marleen de Bruijne, Sarath C. Ranganathan, Harm A.W.M. Tiddens, Stephen M. Stick

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185 Citationer (Scopus)

Abstract

RATIONALE: Chest computed tomography (CT) is the gold standard for demonstrating cystic fibrosis (CF) airways disease. However, there are no standardised outcome measures appropriate for children under 6 years.

OBJECTIVES: We developed the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF), a quantitative measure of airways disease, and compared it to the commonly used CF-CT scoring method.

METHODS: CT scans from the Australian Respiratory Early Surveillance Team for CF (AREST CF) cohort in Western Australia were included. PRAGMA-CF was performed by annotating a grid overlaid on ten axial slices for the presence of bronchiectasis, mucous plugging or other airway abnormalities (inspiratory scans) and trapped air (expiratory scans). The separate proportions of total disease (%Dis), bronchiectasis (%Bx) and trapped air (%TA) were determined. Thirty scans were used for observer reliability, and thirty paired scans obtained at 1 and 3-years old were used for comparison with a validated standard and biological plausibility.

MEASUREMENTS AND MAIN RESULTS: Intraobserver, intraclass correlation coefficients (95% confidence interval) for %Dis, %Bx and %TA were 0.93 (0.86 - 0.97), 0.93 (0.85 - 0.96) and 0.96 (0.91 - 0.98), respectively. The change in %Dis (p = 0.004) and %Bx (p = 0.001) with PRAGMA-CF was related to neutrophil elastase (NE) presence at age 3, whereas only the change in bronchiectasis score was related to NE (p < 0.001) with CF-CT. Sample size calculations for various effect sizes are presented.

CONCLUSIONS: PRAGMA-CF is a sensitive and reproducible outcome measure for assessing the extent of lung disease in very young children with CF.

OriginalsprogEngelsk
TidsskriftAmerican Journal of Respiratory and Critical Care Medicine
Vol/bind191
Udgave nummer10
Sider (fra-til)1158-1165
Antal sider8
ISSN1073-449X
DOI
StatusUdgivet - 2015

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