Study design (if review, criteria of inclusion for studies)
randomized, placebo-controlled parallel design
Participants
children and adults with CF, 181 outpatients
Interventions
10-day administration of three doses of aerosolized rhDNase (0.6, 2.5, or 10.0 mg twice daily)
Outcome measures
efficacy and safety. Forced vital capacity (FVC), forced expiratory volume in one second (FEV1)
Main results
Forced vital capacity (FVC) improved 10 to 12% (p < 0.05 to 0.001), and forced expiratory volume in one second (FEV1) improved 10 to 15% (p < 0.001) across all doses of rhDNase compared with placebo. The magnitude of effect was dose dependent for both FVC and FEV1 through study Day 21 (p < 0.001). rhDNase was associated with a decreased perception of dyspnea and an improved perception of well-being. No patients developed detectable anti-rhDNase antibodies or bronchial reactivity to rhDNase. Some patients experienced mild upper airway irritation, but no major adverse events were reported.
Authors' conclusions
Administration for 10 days of aerosolized rhDNase to pediatric and adult outpatients with CF improves lung function and is well tolerated. Although all three doses were efficacious, the greatest improvement in FEV1 and FEV1/FVC ratio was demonstrated in the 2.5 and 10.0 mg rhDNase treatment groups.