Design and powering of cystic fibrosis clinical trials using pulmonary exacerbation as an efficacy endpoint

D. R. VanDevanter, A. Yegin, W. J. Morgan, S. J. Millar, D. J. Pasta, M. W. Konstan

Research output: Contribution to journalArticlepeer-review

29 Scopus citations


Background: Reduction in pulmonary exacerbations is an important efficacy endpoint for CF clinical studies. Powering exacerbation endpoints requires estimation of the future exacerbation incidence in CF study populations, but rates differ across the population. Methods: We have estimated exacerbation rates for Epidemiologic Study of CF subpopulations stratified by age, FEV1% predicted, sex, weight-for-age percentile, respiratory signs and symptoms, and history of exacerbation and bacterial culture. Sample sizes required to attain 80% power to detect exacerbation reductions of 20% to 80% in 1:1 randomized studies of 3 to 12month duration were determined. Exacerbation treatments with "any" antibiotic (new oral quinolone, new inhaled antibiotic, or intravenous (IV) antibiotic) and with IV antibiotics were studied. Results: At all ages, decreased FEV1, female sex, exacerbation history, and Pseudomonas aeruginosa culture history were associated with increased treatment for exacerbation. Conclusions: These data should assist investigators in the design of future CF exacerbation studies.

Original languageEnglish (US)
Pages (from-to)453-459
Number of pages7
JournalJournal of Cystic Fibrosis
Issue number6
StatePublished - Dec 2011


  • Pulmonary exacerbation
  • Sample size
  • Study design

ASJC Scopus subject areas

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


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