Study design (if review, criteria of inclusion for studies)
randomized, double-blind, placebo- controlled, multicenter study. After the completion of the randomized study, there was an open-label phase
Participants
The first three aerosolisations were performed in the hospital, after bronchial drainage. 70 patients with cystic fibrosis and severe impairment of pulmonary function, aged 5 to 48 years
Interventions
RCT: aerosolised rhDNase 2.5 mg twice daily for 2 weeks (three aerosolisations were performed in the hospital, after bronchial drainage). Open- label phase for 6 months: all patients received rhDNase.
Outcome measures
Respiratory parameters
Main results
Respiratory parameters improved in both groups during the study period. As compared to baseline values, FVC improved by 13.7% and 12.7%, forced expiratory volume in one second (FEV1) by 6% and 7%, respectively in the placebo and rh DNase groups. No significant difference was detected between the two groups. In the open- label phase FEV1 and FVC further improved in all patients.
Authors' conclusions
We failed to demonstrate for severely ill patients with cystic fibrosis a significant advantage for aerosolised rhDNase compared to placebo for two weeks. The further improvement observed in the open-label phase suggests that more prolonged treatment might be necessary to detect a statistically significant benefit. No significant side effects of the treatment were observed.