Study design (if review, criteria of inclusion for studies)
Randomised, open-label, parallel study. Multicentre.
Participants
CF patients (n = 749) with mild lung disease (baseline forced vital capacity less than or equal to70% predicted)
Interventions
dornase alfa 2.5 mg daily for 2 weeks by one of two nebulizer systems: 1) the Medic-Aid Durable SideStream nebulizer powered by the MobilAire Compressor (SS/MA) producing a droplet size with a mass median aerodynamic diameter (MMAD) of 2.1 microm; or 2) the Hudson T Up-draft nebulizer with a DeVilbiss Pulmo-Aide compressor (HT/PA) with an MMAD of 4.9 microm
Outcome measures
Respiratory function (FEV1, FVC and FEF25-75) at 1 and 14 days. Adverse events. Serum levels of dornase alfa at baseline and within 4 hours of the final dose.
Main results
Dornase alfa delivered by both nebulizer systems produced small but statistically significant improvements in pulmonary function compared with baseline. There was a trend (P = 0.06) toward greater improvement in forced expiratory flow in 1 s in the SS/MA group (4.3%) compared with the HT/PA group (2.5%).
Authors' conclusions
These results indicate that the short-term spirometric response to dornase alfa is influenced in part by the physical characteristics of the aerosol in patients with mild lung disease. We speculate that this may be true for other therapeutic aerosols, and it appears that localization of disease in the lung plays a role in the response to inhaled agents.