title: Aerosolized recombinant human DNase in hospitalized cystic fibrosis patients with acute pulmonary exacerbations.
abstract: The goal of this study was to evaluate the safety and efficacy of recombinant human DNase (rhDNase) in hospitalized patients with cystic fibrosis (CF) experiencing acute pulmonary exacerbations. Eighty patients with documented CF were enrolled at 11 CF centers when admitted for antibiotic therapy. Patients were at least 5 yr old with a forced vital capacity (FVC) > or = 35% of predicted and an oxygen saturation > or = 90% on a fraction of inspired oxygen (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 twice daily (n = 37) along with conventional treatment for exacerbations. Administration of rhDNase was not associated with acute adverse events or deaths, and no patients experienced allergic or anaphylactic reactions. Although forced expiratory volume in one second (FEV1) and FVC improved in both treatment groups during the double-blind period, there were no statistically significant differences in the mean change from baseline in FEV1 or FVC between the two groups. rhDNase therapy is safe and well tolerated in CF patients with acute exacerbations requiring hospitalization, but the study did not demonstrate a statistically significant therapeutic effect of rhDNase when added to a regimen of antibiotics and chest physical therapy.
authors.affiliation: Division of Pulmonary Medicine, Children's Hospital Medical Center, Cincinnati, Ohio 45229-3039, USA.
authors.forename: R W
authors.initials: RW
authors.lastname: Wilmott
authors.forename: R S
authors.initials: RS
authors.lastname: Amin
authors.forename: A A
authors.initials: AA
authors.lastname: Colin
authors.forename: A
authors.initials: A
authors.lastname: DeVault
authors.forename: A J
authors.initials: AJ
authors.lastname: Dozor
authors.forename: H
authors.initials: H
authors.lastname: Eigen
authors.forename: C
authors.initials: C
authors.lastname: Johnson
authors.forename: L A
authors.initials: LA
authors.lastname: Lester
authors.forename: K
authors.initials: K
authors.lastname: McCoy
authors.forename: L P
authors.initials: LP
authors.lastname: McKean
authors.forename: R
authors.initials: R
authors.lastname: Moss
authors.forename: M L
authors.initials: ML
authors.lastname: Nash
authors.forename: C P
authors.initials: CP
authors.lastname: Jue
authors.forename: W
authors.initials: W
authors.lastname: Regelmann
authors.forename: D C
authors.initials: DC
authors.lastname: Stokes
authors.forename: H J
authors.initials: HJ
authors.lastname: Fuchs
mesh_terms: D000208:Acute Disease; D000328:Adult; D000336:Aerosols; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000503:physiopathology; D003850:Deoxyribonuclease I / Q000008:administration & dosage / Q000627:therapeutic use*; D004311:Double-Blind Method; D004359:Drug Therapy, Combination; D005100:Expectorants / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D006760:Hospitalization; D006801:Humans; D008297:Male; D010100:Oxygen / Q000097:blood; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D012129:Respiratory Function Tests; D016896:Treatment Outcome
chemical_list: D000336:Aerosols; D005100:Expectorants; D011994:Recombinant Proteins; C085693:DNASE1 protein, human; D003850:Deoxyribonuclease I; D010100:Oxygen