| HOME | HELP | FEEDBACK | SUBSCRIPTIONS | ARCHIVE | SEARCH | TABLE OF CONTENTS |
Guest Access | Sign In via User Name/Password
|
|
|
|||||||
Guest Access | Sign In via User Name/Password |
|||||||||
1 From the Division of Pulmonary Medicine, Children's Hospital, Columbus, Ohio
2 From the Division of Pulmonary Medicine, and Medical Affairs, Genentech, Inc, South San Francisco, Calif.
Study objective: The 12-week efficacy and safety of aerosolized recombinant human DNase (dornase alfa) were evaluated in previously untreated patients with cystic fibrosis (CF) with advanced lung disease.
Design: In this multicenter, double-blind, placebo-controlled study, CF patients with advanced lung disease were randomized to receive either dornase alfa or placebo once a day for 12 weeks.
Patients: A total of 320 patients in clinically stable condition with documented CF and an FVC less than 40% of predicted were recruited from 65 CF Foundation care centers in the United States. The dornase alfa and placebo groups were comparable with respect to age (range, 7 to 57 years), height, and weight. Male subjects outnumbered female subjects (55% vs 45%) and few subjects were younger than 17 years of age (15%). The percentages of predicted FEV1 and FVC were significantly lower in the dornase alfa group at baseline (p
0.05).
Interventions: Patients were randomly assigned to receive either 2.5 mg dornase alfa once daily (n=158) or placebo once daily (n=162). All patients continued to receive standard medications and treatments administered for CF.
Measurements and results: Dornase alfa improved the mean percent change in FEV1 from baseline by 9.4% compared with 2.1% for placebo (p<0.001). The actively treated group showed a 12.4% improvement in FVC compared with 7.3% for placebo (p<0.01). There were no differences between the treatment groups in dyspnea score, number of days receiving IV antibiotics, or length of hospital stay; the overall incidence of adverse events was comparable between treatment groups. Fifteen patients died: 9 in the dornase alfa group and 6 in the placebo group; no differentiating clinical characteristics were demonstrated.
Conclusions: Pulmonary function as measured by FEV1 and FVC improved significantly in the dornase alfa-treated patients. Dornase alfa was found to be safe and well tolerated over the 12-week study period.
Key Words: cystic fibrosis • dornase alfa • pulmonary exacerbation • pulmonary function • recombinant human DNase
Submitted on April 4, 1996
Accepted on May 6, 2007
This article has been cited by other articles:
|
|
 |
|
  R. L. Gibson, J. L. Burns, and B. W. Ramsey Pathophysiology and Management of Pulmonary Infections in Cystic Fibrosis Am. J. Respir. Crit. Care Med., October 15, 2003; 168(8): 918 - 951. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 B. K. Rubin Emerging Therapies for Cystic Fibrosis Lung Disease Chest, April 1, 1999; 115(4): 1120 - 1126. [Full Text] [PDF]
|
|
|
|
 |
|
 J A. INNES DNase in cystic fibrosis: the challenge of assessing response and maximising benefit Thorax, December 1, 1998; 53(12): 1003 - 1004. [Full Text]
|
|
|
|
 |
|
 DORNASE ALFA FOR ADVANCED CYSTIC FIBROSIS LUNG DISEASE Journal Watch (General), November 1, 1996; 1996(1101): 2 - 2. [Full Text]
|
|
| HOME | HELP | FEEDBACK | SUBSCRIPTIONS | ARCHIVE | SEARCH | TABLE OF CONTENTS |
