Cystic fibrosis patients at risk for disease progression marked by decline in FEV1% predicted: development of the cystic fibrosis risk of disease progression score

In the United States cystic fibrosis (CF) is second only to sickle cell anemia as the most common life shortening, recessively inherited disorder typically diagnosed in infancy or early childhood. Approximately 30,000 individuals in the United States have CF with an estimated 1,000 new cases diagnosed each year (1,2). The disease may remain asymptomatic in early childhood, depending on severity. Progression to symptomatic disease state often manifests with mucoviscidosis blocking the airways leading to inflammation with secondary infections and potential for lung damage. Pulmonary exacerbation resulting from disease progression is a major contributor to morbidity and mortality (3-5). Characteristics defining a pulmonary exacerbation include decline in lung function (3,4,6). Limited data describe variables contributory to a sharp decline in FEV1% predicted over a relatively short interval of time; significant lung function decline often results in lung transplantation candidacy or premature death. Determining the phenotypic markers of CF patients likely to experience a significant drop in FEV1% predicted (10 or more percentage points from baseline) will help target efforts for prevention of this deleterious decline. Areas for address may include exposure reduction strategies, therapeutic interventions, and promotion of healthy lifestyle management