A screening tool to identify risk for bronchiectasis progression in
children with cystic fibrosis
Abstract
Background: The marked heterogeneity in CF disease complicates selection
of those most likely to benefit from existing or emergent treatments.
Objective: We aimed to predict progression of bronchiectasis in
preschool children with CF. Methods: Using data collected up to three
years of age, in the Australian Respiratory Early Surveillance Team for
CF (AREST CF) cohort study, clinical information, chest computed
tomography (CT) scores and biomarkers from bronchoalveolar lavage were
assessed in a multivariable linear regression model as predictors for CT
bronchiectasis at age 5-6. Results: Follow-up at 5-6 years was available
in 171 children. Bronchiectasis prevalence at 5-6 was 134/171 (78%) and
median bronchiectasis score 3 (range 0-12). The internally validated
multivariate model retained eight independent predictors accounting for
37% (Adjusted R2) of the variance in bronchiectasis score. The
strongest predictors of future bronchiectasis were: pancreatic
insufficiency, repeated intravenous treatment courses, recurrent lower
respiratory infections in the first 3 years of life and lower airway
inflammation. Dichotomizing the resulting prediction score at a
bronchiectasis score of above the median resulted in a diagnostic odds
ratio of 13 (95% CI 6.3-27) with a positive and negative predictive
values of 80% (95%CI 72%-86%) and 77% (95% CI 69%-83%)
respectively. Conclusion: Early assessment of bronchiectasis risk in
children with CF is feasible with reasonable precision at a group level,
which can assist in high-risk patient selection for interventional
trials. The unexplained variability in disease progression at individual
patient level remains high, limiting the use of this model as a clinical
prediction tool.