Endpoints for clinical trials in young children with cystic fibrosis

Stephanie Davis, Alan S. Brody, Mary J. Emond, Lyndia C. Brumback, Margaret Rosenfeld

Research output: Contribution to journalArticle

66 Citations (Scopus)

Abstract

The availability of sensitive, reproducible, and feasible outcome measures for quantifying lung disease in children with cystic fibrosis (CF) younger than 6 years is critical to the conduct of clinical trials in this important population. Historically, identifying and quantifying the presence of lung disease in very young children with CF was hampered by a lack of reproducible measures of lung function or lung pathology. Over the past 10 years, significant progress has led to physiologic, anatomic, and bronchoscopic measures that may serve as endpoints for future intervention trials. These endpoints include infant and preschool lung function testing, computed tomography of the chest, and bronchoalveolar lavage markers of inflammation and infection. Much progress has occurred in standardizing lung function testing, which is essential for multicenter collaboration. Pulmonary exacerbation has the potential to serve as a clinical endpoint; however, there is currently no standardized definition in children with CF younger than 6 years. Further development of these outcomes measures will enable clinical trials in the youngest CF population with the objective of improving long-term prognosis.

Original languageEnglish (US)
Pages (from-to)418-430
Number of pages13
JournalProceedings of the American Thoracic Society
Volume4
Issue number4
DOIs
StatePublished - Aug 2007
Externally publishedYes

Fingerprint

Cystic Fibrosis
Clinical Trials
Lung
Lung Diseases
Outcome Assessment (Health Care)
Bronchoalveolar Lavage
Population
Thorax
Tomography
Pathology
Inflammation
Infection

Keywords

  • Bronchoalveolar lavage
  • Child
  • Computed tomography scanners
  • Infant
  • Preschool
  • Respiratory function tests
  • X-ray

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Physiology
  • Cell Biology

Cite this

Endpoints for clinical trials in young children with cystic fibrosis. / Davis, Stephanie; Brody, Alan S.; Emond, Mary J.; Brumback, Lyndia C.; Rosenfeld, Margaret.

In: Proceedings of the American Thoracic Society, Vol. 4, No. 4, 08.2007, p. 418-430.

Research output: Contribution to journalArticle

Davis, Stephanie ; Brody, Alan S. ; Emond, Mary J. ; Brumback, Lyndia C. ; Rosenfeld, Margaret. / Endpoints for clinical trials in young children with cystic fibrosis. In: Proceedings of the American Thoracic Society. 2007 ; Vol. 4, No. 4. pp. 418-430.
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