Konstan MW, Rasouliyan L, Pasta DJ, Yegin A, Morgan WJ, Wagener JS, Scientific Advisory, Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Trends in the use of routine therapies in cystic fibrosis: 1995-2005. Pediatr Pulmonol. 2010 Dec;45(12):1167-72. doi: 10.1002/ppul.21315.


Many therapies are used to treat manifestations of cystic fibrosis (CF). Trends in routine therapy use in Epidemiologic Study of Cystic Fibrosis patients were studied from 1995 to 2005. Patients (15,087) were assessed in 1995; 12,778 in 2005. Observed differences in therapy use of greater than or equal 2% were statistically significant at P less than  0.001. Comparing the 1995 and 2005 populations, mean age was 13.9 versus 15.5 years; weight-for-age percentile was 30.3 versus 36.9; and mean forced expiratory volume in 1 sec (FEV(1)) was 73.7% (n = 7065) versus 78.7% (n = 7867) predicted. Use of several therapies increased, including airway clearance (69.9-89.6%), inhaled bronchodilators (72.0-84.0%), dornase alfa (44.8-67.2%), inhaled corticosteroids (16.0-49.3%), inhaled antibiotics (6.5-43.1%), oral nutritional supplements (18.3-24.5%), and insulin/oral hypoglycemic agents (4.9-10.2%). Use of mast cell stabilizers (from 22.0% to 5.3%) and oral bronchodilators (from 10.4% to 1.5%) decreased. Less dramatic changes occurred for pancreatic enzymes (92.6-91.0%), oral nonquinolone antibiotics (44.7-39.8%), oral corticosteroids (7.8-5.2%), mucolytics (4.4-2.5%), NSAIDs/high-dose ibuprofen (3.6-3.3%), enteral nutrition (5.2% vs. 8.2%), and oxygen (4.7-4.5%). Therapies not tracked in 1995 were evident in 2005, including oral macrolide antibiotics (33.8%), leukotriene inhibitors/antagonists (10.8%), and inhaled hypertonic saline (2.6%). Routine therapies were generally used more often by older patients and those with lower FEV(1). Notable increases in use of therapies, particularly of inhaled therapies, suggest that overall patient treatment burden must have risen correspondingly.

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