4.6 Article

The effect of dornase alfa on ventilation inhomogeneity in patients with cystic fibrosis

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

  1. Roche Canada
  2. Lynn and Arnold Irwin Foundation

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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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