RT Journal
A1 Dasenbrook EC, Konstan MW
T1 INhaled hypertonic saline in infants and young children with cystic fibrosis
JF JAMA
JO JAMA
YR 2012
FD June 6
VO 307
IS 21
SP 2316
OP 2317
DO 10.1001/jama.2012.5853
UL http://dx.doi.org/10.1001/jama.2012.5853
AB 
Cystic fibrosis is caused by a mutation in the gene that encodes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.3 Absence of functional CFTR at the surface of the airway epithelium in a patient with CF results in decreased chloride secretion and increased sodium absorption, which causes depletion of water from the airway surface. This results in impaired mucociliary clearance, leading to a favorable environment for infection and inflammation to occur, and ultimately leads to lung destruction.3 Thus, restoring salt and water balance in the airways of patients with CF has been the goal of a number of therapeutic strategies. In a recent clinical trial, ivacaftor, a potentiator of CFTR, was shown to improve lung function and decrease pulmonary exacerbations.4 Although ivacaftor targets a specific mutation affecting only 4% to 5% of patients with CF worldwide, other CFTR-specific investigational agents that have the potential to affect nearly all patients with CF are currently being studied. In the meantime, adding salt directly to the airway may be of benefit, and several studies have demonstrated the efficacy of this approach in patients older than 6 years with CF.5- 6