Trends in the use of routine therapies in cystic fibrosis: 1995-2005

Abstract

Summary 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 through 2005. 15,087 patients were assessed in 1995; 12,778 in 2005. Observed differences in therapy use of ≥2% were statistically significant at P < 0.001. Comparing the 1995 and 2005 populations, mean age was 13.9 vs. 15.5 years; weight-for-age percentile was 30.3 vs. 36.9; and mean forced expiratory volume in 1 s (FEV1) was 73.7 (n = 7065) vs. 78.7 (n = 7867) percent predicted. Use of several therapies increased, including airway clearance (69.9 to 89.6%), inhaled bronchodilators (72.0 to 84.0%), dornase alfa (44.8 to 67.2%), inhaled corticosteroids (16.0 to 49.3%), inhaled antibiotics (6.5 to 43.1%), oral nutritional supplements (18.3 to 24.5%), and insulin/oral hypoglycemic agents (4.9 to 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 to 91.0%), oral nonquinolone antibiotics (44.7 to 39.8%), oral corticosteroids (7.8 to 5.2%), mucolytics (4.4 to 2.5%), NSAIDs/high-dose ibuprofen (3.6 to 3.3%), enteral nutrition (5.2 vs. 8.2%), and oxygen (4.7 to 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 FEV1. Notable increases in use of therapies, particularly of inhaled therapies, suggest that overall patient treatment burden must have risen correspondingly.

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

“…These mutations result in a lack of functional CFTR protein or dysfunctional CFTR protein channels at the surface of epithelial cells, which causes impaired chloride transport, dysregulated fluid balance, and thickened mucosal secretions in organ systems such as the lungs, pancreas, sweat glands, and reproductive organs [1,16]. Most available treatments for CF address the symptoms and sequelae of the disease rather than the underlying molecular pathophysiology [8,11].…”

Carrier-free high-dose dry powder inhaler formulation of ibuprofen: Physicochemical characterization and in vitro aerodynamic performance

“…Even though IBU holds promise in the management of CF, the use of this treatment modality has not been well-adopted or widespread. From 1995 to 2005, the frequency of use of oral, high-dose IBU remained low (3.6 vs. 3.3 %) according to an epidemiologic study of CF patients (Konstan et al, 2010). High doses, side effects, contraindications, and black box warnings regarding the use of IBU in patients with a moderate to severe renal impairment, or with an increased risk of upper gastrointestinal (GI) bleeding (Bhala et al, 2013;Lexi-Comp, 2015), may be responsible for this apparent under-prescribing and underuse in this patient population.…”

Safety, Tolerability, and Plasma Exposure of Tiotropium Respimat® in Children and Adults with Cystic Fibrosis

“…(2,3) More than 80% of patients with CF are prescribed short-or long-acting bronchodilators; b-agonists are the most widely used agents, and anticholinergics are used to a much lesser extent. (4) However, none of these agents are specifically approved for use in CF and their therapeutic role has not been clearly defined. (5) The available evidence indicates that bronchodilator agents are used in CF to relieve airway obstruction and improve airway clearance, leading to improved lung function.…”