Measuring and improving respiratory outcomes in cystic fibrosis lung disease: opportunities and challenges to therapy.
Cystic fibrosis (CF) is a life-shortening disease with significant morbidity. Despite overall improvements in survival, patients with CF experience frequent pulmonary exacerbations and declining lung function, which often accelerates during adolescence. New treatments target steps in the pathogenesis of lung disease, such as the basic defect in CF (CF Transmembrane Conductance Regulator [CFTR]), pulmonary infections, inflammation, and mucociliary clearance. These treatments offer hope but also present challenges to patients, clinicians, and researchers. Comprehensive assessment of efficacy is critical to identify potentially beneficial treatments. Lung function and pulmonary exacerbation are the most commonly used outcome measures in CF clinical research. Other outcome measures under investigation include measures of CFTR function; biomarkers of infection, inflammation, lung injury and repair; and patient-reported outcomes. Molecular diagnostics may help elucidate the complex CF airway microbiome. As new treatments are developed for patients with CF, efforts should be made to balance treatment burden with quality of life. This review highlights emerging treatments, obstacles to optimizing outcomes, and key future directions for research.
Zemanick
Edith T
ET
Department of Pediatrics, University of Colorado Denver, 13123 E. 16th Avenue, Aurora, CO 80045, USA. zemanick.edith@tchden.org <zemanick.edith@tchden.org>
Harris
J Kirk
JK
Conway
Steven
S
Konstan
Michael W
MW
Marshall
Bruce
B
Quittner
Alexandra L
AL
Retsch-Bogart
George
G
Saiman
Lisa
L
Accurso
Frank J
FJ
D000900:Anti-Bacterial Agents / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000401:mortality*; D015316:Genetic Therapy; D006801:Humans; D009017:Morbidity; D018410:Pneumonia, Bacterial / Q000188:drug therapy* / Q000401:mortality*
D000900:Anti-Bacterial Agents