Document Detail

Multicenter evaluation of infant lung function tests as cystic fibrosis clinical trial endpoints.
MedLine Citation:
PMID:  20622043     Owner:  NLM     Status:  MEDLINE    
RATIONALE: The conducting of clinical trials in infants with cystic fibrosis (CF) has been hindered by lack of sensitive outcome measures.
OBJECTIVES: To evaluate safety, feasibility, and ability to detect abnormalities in lung function of serial pulmonary function tests (PFTs) in infants with CF.
METHODS: Multicenter observational study using a commercial device, rigorous training, ongoing quality control, and over-reading of data by an independent panel. Raised volume rapid thoracoabdominal compression technique and plethysmography were performed at enrollment and at 6 and 12 months, with an additional 1-month reproducibility visit.
MEASUREMENTS AND MAIN RESULTS: A total of 342 procedures were performed in 100 infants with CF at 10 centers. FRC measurements were acceptable at a higher proportion of study visits (89%) than raised volume (72%) or fractional lung volume (68%) measurements. Average Z scores for many parameters differed significantly from historical control values. Mean (95% confidence interval) Z scores were: -0.52 (-0.78 to -0.25) for forced expiratory flow at 75% (FEF₇₅) for FVC; 1.92 (1.39-2.45) for FRC; 1.22 (0.68-1.76) for residual volume; 0.87 (0.60-1.13) for FRC/total lung capacity; and 0.66 (0.27-1.06) for residual volume/total lung capacity. For future multicenter clinical trials using infant PFTs as primary endpoints, minimum detectable treatment effects are presented for several sample sizes.
CONCLUSIONS: In this 10-center study, key PFT measures were significantly different in infants with CF than in historical control subjects. However, infant PFTs do not yet appear ready as primary efficacy endpoints for multicenter clinical trials, particularly at inexperienced sites, based on acceptability rates, variability, and potentially large sample sizes required to detect reasonable treatment effects.
Stephanie D Davis; Margaret Rosenfeld; Gwendolyn S Kerby; Lyndia Brumback; Margaret H Kloster; James D Acton; Andrew A Colin; Carol K Conrad; Meeghan A Hart; Peter W Hiatt; Peter J Mogayzel; Robin C Johnson; Stephanie L Wilcox; Robert G Castile
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Publication Detail:
Type:  Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't     Date:  2010-07-09
Journal Detail:
Title:  American journal of respiratory and critical care medicine     Volume:  182     ISSN:  1535-4970     ISO Abbreviation:  Am. J. Respir. Crit. Care Med.     Publication Date:  2010 Dec 
Date Detail:
Created Date:  2010-12-02     Completed Date:  2011-02-11     Revised Date:  2013-05-29    
Medline Journal Info:
Nlm Unique ID:  9421642     Medline TA:  Am J Respir Crit Care Med     Country:  United States    
Other Details:
Languages:  eng     Pagination:  1387-97     Citation Subset:  AIM; IM    
Department of Pediatrics, North Carolina Children’s Hospital, University of North Carolina at Chapel Hill, USA.
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MeSH Terms
Clinical Trials as Topic*
Cystic Fibrosis / diagnosis*
Feasibility Studies
Forced Expiratory Volume
Longitudinal Studies
Plethysmography / methods
Prospective Studies
Reproducibility of Results
Respiratory Function Tests / methods
Sensitivity and Specificity
Comment In:
Am J Respir Crit Care Med. 2010 Dec 1;182(11):1340-2   [PMID:  21123748 ]

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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