PT  - JOURNAL ARTICLE
AU  - R. Amin
AU  - P. Subbarao
AU  - W. Lou
AU  - A. Jabar
AU  - S. Balkovec
AU  - R. Jensen
AU  - S. Kerrigan
AU  - P. Gustafsson
AU  - F. Ratjen
TI  - The effect of dornase alfa on ventilation inhomogeneity in patients with cystic fibrosis
AID  - 10.1183/09031936.00072510
DP  - 2010 Jan 01
TA  - European Respiratory Journal
PG  - erj00725-2010
4099  - http://erj.ersjournals.com/content/early/2010/08/06/09031936.00072510.short
4100  - http://erj.ersjournals.com/content/early/2010/08/06/09031936.00072510.full
AB  - 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 and 18 years of age with FEV1% ≥80% predicted were eligible. In a crossover design, 17 patients received 4 weeks of dornase alfa and placebo in a randomized sequence separated by a 4-weekwashout period. The primary endpoint was the change in LCI from dornase alfa versus placebo. A mixed model approach incorporating period-dependent baselines was used. This trial was registered with ClinicalTrials.gov, number NCT00557089.The mean age±SD was 10.32±3.35 years. Dornase alfa improved LCI versus placebo (0.90±1.44, p=0.022). A significant improvement in LCI occurred in seven of the seventeen subjects. Small airways flows as measured by forced expiratory flow at 25–75% expired volume (FEF25–75) measured by percent predicted and z-scores also improved in subjects on dornase alfa (6.1%±10.34, p=0.03; and 0.28 z-score±0.46, 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.