Cystic fibrosis research
People with cystic fibrosis (CF) inherit a defective gene called CFTR (CF transmembrane conductance regulator).
Ivacaftor is a CFTR gene-potentiating agent licensed for use in patients with Class III gating CF mutations. The beneficial effects on lung function, exacerbation rate, rate of hospitalisation and quality of life indices have been well established. To date, however, the effects on exercise capacity and other indices of extra-pulmonary health are unknown.
With this in mind the CF Service at The Alfred undertook a double-blind, placebo controlled trial in 20 adult patients with CF. At the end of the study period all participants commenced open-label medication and had a further assessment at 24 weeks post commencement.
Participants underwent six assessments involving cardiopulmonary exercise testing, spirometry, bloods, sputum
samples, quality of life assessments and sweat tests. The primary end-point for the study was VO2max.
Secondary endpoints included FEV1 percent predicted, sweat chloride levels, inflammatory markers, quality of life indices and changes in the sputum microbiome (analysis on genetically distinct molecular species). Sputum samples were examined using cDNA microbiome analysis techniques.