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PRAGMA-CF. A Quantitative Structural Lung Disease Computed Tomography Outcome in Young Children with Cystic Fibrosis

Chest computed tomography (CT) is the gold standard for demonstrating cystic fibrosis (CF) airway disease. However, there are no standardized outcome...

Authors:
Rosenow T, Oudraad MC, Murray CP, Turkovic L, Kuo W, de Bruijne M, ... Stick SM.

Authors notes:
American journal of respiratory and critical care medicine. 2015;191(10):1158-65.

Keywords:
clinical trials as topic, medical imaging, pediatrics, Chest computed tomography, cystic fibrosis

Abstract:
RATIONALE: Chest computed tomography (CT) is the gold standard for demonstrating cystic fibrosis (CF) airway disease.

However, there are no standardized outcome measures appropriate for children younger than 6 years.

OBJECTIVES: We developed the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF), a quantitative measure of airway disease, and compared it with 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 10 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 30 paired scans obtained at 1 and 3 years old were used for comparison with a validated standard and biologic plausibility.

MEASUREMENTS AND MAIN RESULTS: Intraobserver, intraclass correlation coefficients for %Dis, %Bx, and %TA were 0.93, 0.93, and 0.96, respectively.

The change in %Dis and %Bx with PRAGMA-CF was related to neutrophil elastase presence at age 3, whereas only the change in bronchiectasis score was related to neutrophil elastase 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.