Document Detail


Design and powering of cystic fibrosis clinical trials using pulmonary exacerbation as an efficacy endpoint.
MedLine Citation:
PMID:  21803665     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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, FEV(1)% 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 12 month 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 FEV(1), 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.
Authors:
D R Vandevanter; A Yegin; W J Morgan; S J Millar; D J Pasta; M W Konstan
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Publication Detail:
Type:  Journal Article     Date:  2011-07-30
Journal Detail:
Title:  Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society     Volume:  10     ISSN:  1873-5010     ISO Abbreviation:  J. Cyst. Fibros.     Publication Date:  2011 Dec 
Date Detail:
Created Date:  2011-11-21     Completed Date:  2012-04-02     Revised Date:  2014-07-04    
Medline Journal Info:
Nlm Unique ID:  101128966     Medline TA:  J Cyst Fibros     Country:  Netherlands    
Other Details:
Languages:  eng     Pagination:  453-9     Citation Subset:  IM    
Copyright Information:
Copyright © 2011 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Child
Clinical Trials as Topic / statistics & numerical data*
Cystic Fibrosis / physiopathology*,  therapy
Disease Progression
Female
Forced Expiratory Volume*
Humans
Lung / physiopathology*
Male
Research Design / statistics & numerical data*
Sample Size
Treatment Outcome
Young Adult
Grant Support
ID/Acronym/Agency:
P30 DK027651/DK/NIDDK NIH HHS
Comments/Corrections

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