Measuring and improving respiratory outcomes in cystic fibrosis lung disease: Opportunities and challenges to therapy

Edith T. Zemanick, J. Kirk Harris, Steven Conway, Michael W. Konstan, Bruce Marshall, Alexandra Quittner, George Retsch-Bogart, Lisa Saiman, Frank J. Accurso

Research output: Contribution to journalArticle

79 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)1-16
Number of pages16
JournalJournal of Cystic Fibrosis
Volume9
Issue number1
DOIs
StatePublished - Jan 1 2010

Fingerprint

Cystic Fibrosis
Lung Diseases
Lung
Therapeutics
Outcome Assessment (Health Care)
Mucociliary Clearance
Cystic Fibrosis Transmembrane Conductance Regulator
Molecular Pathology
Microbiota
Lung Injury
Infection
Research
Pneumonia
Biomarkers
Quality of Life
Research Personnel
Inflammation
Morbidity
Survival

Keywords

  • Cystic fibrosis
  • Emerging treatments
  • Improved outcomes

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Pediatrics, Perinatology, and Child Health

Cite this

Zemanick, E. T., Harris, J. K., Conway, S., Konstan, M. W., Marshall, B., Quittner, A., ... Accurso, F. J. (2010). Measuring and improving respiratory outcomes in cystic fibrosis lung disease: Opportunities and challenges to therapy. Journal of Cystic Fibrosis, 9(1), 1-16. https://doi.org/10.1016/j.jcf.2009.09.003

Measuring and improving respiratory outcomes in cystic fibrosis lung disease : Opportunities and challenges to therapy. / Zemanick, Edith T.; Harris, J. Kirk; Conway, Steven; Konstan, Michael W.; Marshall, Bruce; Quittner, Alexandra; Retsch-Bogart, George; Saiman, Lisa; Accurso, Frank J.

In: Journal of Cystic Fibrosis, Vol. 9, No. 1, 01.01.2010, p. 1-16.

Research output: Contribution to journalArticle

Zemanick, ET, Harris, JK, Conway, S, Konstan, MW, Marshall, B, Quittner, A, Retsch-Bogart, G, Saiman, L & Accurso, FJ 2010, 'Measuring and improving respiratory outcomes in cystic fibrosis lung disease: Opportunities and challenges to therapy', Journal of Cystic Fibrosis, vol. 9, no. 1, pp. 1-16. https://doi.org/10.1016/j.jcf.2009.09.003
Zemanick, Edith T. ; Harris, J. Kirk ; Conway, Steven ; Konstan, Michael W. ; Marshall, Bruce ; Quittner, Alexandra ; Retsch-Bogart, George ; Saiman, Lisa ; Accurso, Frank J. / Measuring and improving respiratory outcomes in cystic fibrosis lung disease : Opportunities and challenges to therapy. In: Journal of Cystic Fibrosis. 2010 ; Vol. 9, No. 1. pp. 1-16.
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