TY - JOUR
T1 - Measuring and improving respiratory outcomes in cystic fibrosis lung disease
T2 - Opportunities and challenges to therapy
AU - Zemanick, Edith T.
AU - Harris, J. Kirk
AU - Conway, Steven
AU - Konstan, Michael W.
AU - Marshall, Bruce
AU - Quittner, Alexandra L.
AU - Retsch-Bogart, George
AU - Saiman, Lisa
AU - Accurso, Frank J.
N1 - Funding Information:
Edith Zemanick has nothing to disclose relevant to this manuscript. J. Kirk Harris has nothing to disclose relevant to this manuscript. Steven Conway is a member of the advisory board for Phillips Medizinsysteme Böblingen (Germany) and has recently served on an advisory board for Gilead Sciences, Inc. He has previously served on advisory boards for Roche and Novartis Pharmaceuticals. Michael W. Konstan has served as a paid consultant to Axcan Pharma, Digestive Care Inc, Genentech, Gilead Sciences, Inc., Novartis Pharmaceuticals, PTC Therpaeutics, Solvay, Transave, Inc., and Vertex Pharmaceuticals, Inc. Bruce Marshall has nothing to disclose relevant to this manuscript. Alexandra L. Quittner has received consulting income from Gilead Sciences, Inc., Novartis Pharmaceuticals, Vertex Pharmaceuticals, Inc., and Transave, Inc. She serves on the North American Advisory Group for Genentech (NASAG) and has an investigator-initiated grant from Novartis Pharmaceuticals. George Retsch-Bogart received support as a site principal investigator to conduct clinical trials in the Cystic Fibrosis Foundation Therapeutics Development Network from Gilead Sciences, Inc. and Inspire Pharmaceuticals. Lisa Saiman has served on advisory boards for Aridis Pharmaceuticals, Gilead Sciences, Inc., Novartis Pharmaceuticals, and Transave, Inc. She has received research funding from Bayer and Chiesi Pharmaceuticals and has served on the Cystic Fibrosis Foundations Data Safety Monitoring Board. Frank Accurso has no personal conflict of interest. Dr. Accurso's institution received funding for clinical trials for agents mentioned in this manuscript from Gilead Sciences, Inc., Vertex Pharmaceuticals, Inspire Pharmaceuticals and PTC Therapeutics.
PY - 2010/1
Y1 - 2010/1
N2 - 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.
AB - 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.
KW - Cystic fibrosis
KW - Emerging treatments
KW - Improved outcomes
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U2 - 10.1016/j.jcf.2009.09.003
DO - 10.1016/j.jcf.2009.09.003
M3 - Review article
C2 - 19833563
AN - SCOPUS:73449113631
SN - 1569-1993
VL - 9
SP - 1
EP - 16
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 1
ER -