AEROSOLIZED RECOMBINANT HUMAN DNASE IN HOSPITALIZED CYSTIC-FIBROSIS PATIENTS WITH ACUTE PULMONARY EXACERBATIONS

The goal of this study was to evaluate the safety and efficacy of reco mbinant human DNase (rhDNase) in hospitalized patients with cystic fib rosis (CF) experiencing acute pulmonary exacerbations. Eighty patients with documented CF were enrolled at 11 CF centers when admitted for a ntibiotic therapy. Patients were at least 5 yr old with a forced vital capacity (FVC) greater than or equal to 35% of predicted and an oxyge n saturation greater than or equal to 90% on a fraction of inspired ox ygen (FIO2) < 0.5. Patients were randomized to receive rhDNase 2.5 mg in 2.5 ml excipient twice a day (n = 43) or 2.5 ml excipient alone twi ce daily (n = 37) along with conventional treatment for exacerbations. Administration of rhDNase was not associated with acute adverse event s or deaths, and no patients experienced allergic or anaphylactic reac tions. Although forced expiratory volume in one second (FEV(1)) and NC improved in both treatment groups during the double-blind period, the re were no statistically significant differences in the mean change fr om baseline in FEV(1) or NC between the two groups. rhDNase therapy is safe and well tolerated in CF patients with acute exacerbations requi ring hospitalization, but the study did not-demonstrate a statisticall y significant therapeutic effect of rhDNase when added to a regimen of antibiotics and chest physical therapy.