ABSTRACT
Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and â¼75,000 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. The ultimate goal of therapy will be to deliver tailored individualized therapies to those who need it the most. This review will focus on new research on CF transmembrane conductance regulator protein modulator agents and the effort of gene therapy.
ABSTRACT
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. The primary end point was the change in the LCI due to HS versus IS. RESULTS Baseline characteristics including the LCI were not significantly different between both study periods. Four weeks of twice-daily HS inhalation significantly improved the LCI compared with IS (1.16, 95% CI 0.26 to 2.05; p=0.016), whereas other outcome measures such as spirometry and quality of life failed to reach statistical significance. Randomisation order had no significant impact on the treatment effect. CONCLUSIONS The LCI, but not spirometry was able to detect a treatment effect from HS inhalation in patients with CF with mild disease and may be a suitable tool to assess early intervention strategies in this patient population. Clinical trial number NCT00635141.
