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 L. Quittner, George Retsch-Bogart, Lisa Saiman, Frank J. Accurso

Research output: Contribution to journalReview articlepeer-review

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

Keywords

  • Cystic fibrosis
  • Emerging treatments
  • Improved outcomes

ASJC Scopus subject areas

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

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