Original ArticlesDefining a pulmonary exacerbation in cystic fibrosis☆,☆☆
Section snippets
Study design
Physician-investigators completed a PEx questionnaire at 3 scheduled visits for each subject participating in a multicenter, phase III, randomized, controlled trial of tobramycin for inhalation.6 Subjects enrolled in this trial received inhaled tobramycin or placebo twice daily for 28 days, followed by 28 days without study drug. This cycle was repeated 2 more times during the 6-month study period. Treatment with conventional therapy, including oral and intravenous antibiotics, was not altered
Subject characteristics
A total of 520 subjects were enrolled in the clinical trial (258 in the tobramycin group and 262 in the placebo group). PEx data were available from 246 subjects in the placebo arm at the week 8 study visit (94% of total subjects enrolled in the placebo group) and from 234 subjects at the week 20 visit (89% of subjects in the placebo group). Subjects in the current study had demographic characteristics similar to those of subjects enrolled in the phase III study as a whole (Table I).They were
Discussion
The goal of the current study was to develop a standardized definition of a PEx for CF clinical trials that did not rely on therapeutic decisions. We evaluated the assessments of more than 100 physicians as to whether subjects were experiencing a PEx to determine the key components of their decision-making processes. We found that the features most important in determining the presence of a PEx were symptoms and features of the clinical history. Chest examination findings and pulmonary function
Acknowledgements
We thank the investigators in the Cystic Fibrosis Inhaled Tobramycin Study Group (seeAppendix 2).
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Cited by (0)
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Supported by the Cystic Fibrosis Foundation (Bethesda, Md) and PathoGenesis Corporation (Seattle, Wash).
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Reprint requests: Margaret Rosenfeld, MD, MPH, Division of Pulmonary Medicine, Children's Hospital and Regional Medical Center, 4800 Sandpoint Way, Seattle, WA 98105.