期刊
EUROPEAN RESPIRATORY JOURNAL
卷 37, 期 4, 页码 806-812出版社
EUROPEAN RESPIRATORY SOC JOURNALS LTD
DOI: 10.1183/09031936.00072510
关键词
Children; cystic fibrosis; dornase alfa; lung clearance index; spirometry; ventilation inhomogeneity
资金
- Roche Canada
- Lynn and Arnold Irwin Foundation
Outcome measures to assess therapeutic interventions in cystic fibrosis (CF) patients with mild lung disease are lacking. Our aim was to determine if the lung clearance index (LCI) can detect a treatment response to dornase alfa in paediatric CF patients with normal spirometry. CF patients between 6-18 yrs of age with FEV1 >= 80% pred were eligible. In a crossover design, 17 patients received 4 weeks of dornase alfa and placebo in a randomised sequence separated by a 4-week washout period. The primary end-point was the change in LCI from dornase alfa versus placebo. A mixed model approach incorporating period-dependent baselines was used. The mean +/- SD age was 10.32 +/- 3.35 yrs. Dornase alfa improved LCI versus placebo (0.90 +/- 1.44; p=0.022). Forced expiratory flow at 25-75% expired volume measured by % pred and z-scores also improved in subjects on dornase alfa (6.1%+/- 10.34%; p=0.03 and 0.28 +/- 0.46 z-score; p=0.03). Dornase alfa significantly improved LCI. Therefore the LCI may be a suitable tool to assess early intervention strategies in this patient population.
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