Abstract
Better treatment of obstructed small airways is needed in cystic fibrosis. This study investigated whether efficient deposition of dornase alfa in the small airways improves small airway obstruction.
In a multicentre, double-blind, randomised controlled clinical trial, cystic fibrosis patients on maintenance treatment with 2.5 mL dornase alfa once daily were switched to a smart nebuliser and randomised to small airway deposition (n=24) or large airway deposition (n=25) for 4 weeks. The primary outcome parameter was forced expiratory flow at 75% of forced vital capacity (FEF75%).
FEF75% increased significantly by 0.7 sd (5.2% predicted) in the large airways group and 1.2 sd (8.8% pred) in the small airways group. Intention-to-treat analysis did not show a significant difference in treatment effect between groups. Per-protocol analysis, excluding patients not completing the trial or with adherence <70%, showed a trend (p=0.06) in FEF75% Z-score and a significant difference (p=0.04) between groups in absolute FEF75% (L·s−1) favouring small airway deposition.
Improved delivery of dornase alfa using a smart nebuliser that aids patients in correct inhalation technique resulted in significant improvement of FEF75% in children with stable cystic fibrosis. Adherent children showed a larger treatment response for small airway deposition.
Footnotes
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Clinical Trial
This study is registered at www.trialregister.nl and in the International Standard Randomised Controlled Trial Number Register with identifier number 6422851.
Statement of Interest
Statements of interest for E.M. Bakker, B.M. Assael and H.A.W.M. Tiddens, and for the study itself, can be found at www.erj.ersjournals.com/site/misc/statements.xhtml
- Received January 13, 2011.
- Accepted May 14, 2011.
- ©ERS 2011