New and Evolving Therapies for Cystic Fibrosis Patients.

Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and ∼75,000 people in North America, Europe, and Australia. The purpose of this review is to discuss the most recent advances in therapies for CF patients. The CF drug development pipeline provides information regarding the phase of each new therapy and those that are already in use by patients.

Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function.

BACKGROUND AND AIMS Sensitive outcome measures to assess the efficacy of therapeutic interventions in patients with cystic fibrosis (CF) with mild lung disease are currently lacking. Our objective was to study the ability of the lung clearance index (LCI), a measure of ventilation inhomogeneity, to detect a treatment response to hypertonic saline inhalation in paediatric patients with CF with normal spirometry. METHODS In a crossover trial, 20 patients with CF received 4 weeks of hypertonic saline (HS) and isotonic saline (IS) in a randomised sequence separated by a 4 week washout period.