Phenotype of Rapid Cystic Fibrosis Lung Disease Progression During Adolescence and Young Adulthood

Cystic fibrosis (CF) causes progressive lung damage through chronic mucus obstruction, infection and inflammation. This pathologic cycle leads to lung function decline that typically is manifest during adolescence. Understanding the factors that predict those at highest risk of lung function decline would help treating physicians identify, and target, those patients most likely to benefit from available interventions. In the July 2017 issue of the American Journal of Respiratory and Critical Care Medicine, Dr. Rhonda Szczesniak, PhD, in the Division of Biostatistics and Epidemiology, and Dr. J.P. Clancy, MD, in the Division of Pulmonary Medicine, teamed up to examine this question using the Cystic Fibrosis Foundation Patient Registry (CFFPR). This database includes longitudinal clinical information from ~30,000 patients in the U.S. Using novel statistical modeling tools, the team identified critical determinants of lung function decline. First, patients tended to cluster into three groups consisting of early, middle and late onset of lung function decline, spanning early to late adolescence. They did not differ substantially by the rate of lung function loss, but rather the timing of onset. Second, those with a childhood history of frequent pulmonary exacerbations, and low initial lung function, were at highest risk of early decline. Highlighted impact of this work in an accompanying editorial stated, "Ultimately further understanding of the biologic and social determinants underpinning divergent CF lung disease phenotypes is needed if the full potential of precision medicine for treatment of CF is to be realized."

Szczesniak RD, Li D, Su W, Brokamp C, Pestian J, Seid M, Clancy JP. Phenotypes of Rapid Cystic Fibrosis Lung Disease Progression during Adolescence and Young Adulthood. Am J Respir Crit Care Med. 2017 Aug 15;196(4):471-478.