CFF Biomarkers of Exacerbation

Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of
biomarkers that can be used to identify promising new therapies. There is an urgent need for
development and validation of biomarkers that more quickly predict the usefulness of
potential drugs in CF and might prognosticate clinical course. In particular, combinations
of protein biomarkers that can be obtained non-invasively offer great promise. The goal of
this project is to determine whether protein biomarkers in blood can demonstrate a
beneficial effect of treatment over two weeks. We intend to initially target an acute
pulmonary exacerbation in CF because we know that subjects being treated with intravenous
antibiotics and enhanced mucus clearance display clinical improvements within two weeks. We
propose to prospectively collect blood samples from a large cohort of well-characterized CF
persons serially during inpatient admissions for a pulmonary exacerbation and longitudinally
during annual visits. Through this proposal, we hope to identify a CF lung injury biomarker
panel that increases in the setting of an acute pulmonary exacerbation and improves rapidly
following intravenous antibiotic therapy. Additionally, we will begin to explore whether
this CF lung injury biomarker panel might also prognosticate clinical course including
decline in pulmonary function. Finally, this study will serve as an important source of
blood samples that will be banked for future biomarker and therapeutic studies designed to
benefit the entire CF community.