Recombinant human DNase I in cystic fibrosis patients with severe pulmonary disease: a short-term, double-blind study followed by six months open-label treatment.
70 most severly ill CF patients (FVC < 40% predicted) were recruited for the double-blind study, and 64 entered the OEP of whom 38 completed.
Interventions
2.5 mg rhDNase twice daily or placebo for a period of 14 days followed by a 6 month open extension period (OEP). During the OEP, all patients received 2.5 mg rhDNase twice daily.
Outcome measures
safety and efficacy of rhDNase
Main results
In both the double-blind period and the OEP the drug appeared to be safe. During the double-blind study, forced expiratory volume in one second (FEV1) and FVC improved in both groups but there was no statistically significant difference between the groups. In the OEP, there was mean improvement in percentage predicted FEV1 and FVC, 9 and 18%, respectively, for all patients participating.
Authors' conclusions
In conclusion, DNase is safe when administered in conjunction with a rigorous regimen of chest physiotherapy to severely ill patients (FVC < 40% predicted) with CF.