RT Journal Article
SR Electronic
T1 Improved treatment response to dornase alfa in cystic fibrosis patients using controlled inhalation
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP erj00062-2011
DO 10.1183/09031936.00006211
A1 E.M. Bakker
A1 S. Volpi
A1 E. Salonini
A1 E.C. van der Wiel – Kooij
A1 C.J.J.C.M. Sintnicolaas
A1 W.C.J. Hop
A1 B.M. Assael
A1 P.J.F.M. Merkus
A1 H.A.W.M. Tiddens
YR 2011
UL http://erj.ersjournals.com/content/early/2011/07/07/09031936.00006211.abstract
AB Better treatment of obstructed small airways is needed in CF. This study investigated whether efficient deposition of dornase alfa in the small airways improves small airway obstruction.In a multi-centre, double-blind, randomized controlled clinical trial, CF patients on maintenance treatment with 2.5 mL dornase alfa once daily were switched to a smart nebulizer and randomized to small airways deposition (n=24) or large airways 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% predicted) 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 airways deposition.Improved delivery of dornase alfa using a smart nebulizer that aids patients in correct inhalation technique resulted in significant improvement of FEF75 in children with stable CF. Adherent children showed a larger treatment response for small airways deposition.