RT Journal Article
SR Electronic
T1 The effect of dornase alfa on ventilation inhomogeneity in patients with cystic fibrosis
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP erj00725-2010
DO 10.1183/09031936.00072510
A1 R. Amin
A1 P. Subbarao
A1 W. Lou
A1 A. Jabar
A1 S. Balkovec
A1 R. Jensen
A1 S. Kerrigan
A1 P. Gustafsson
A1 F. Ratjen
YR 2010
UL http://erj.ersjournals.com/content/early/2010/08/06/09031936.00072510.abstract
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.