Study design (if review, criteria of inclusion for studies)
Randomised, double-blind, parallel design trial over 12 weeks.
Participants
40 participants withdrew from the trial, five due to adverse events, 10 withdrew consent, 1 did not comply with the study protocol, 15 died, 2 were unavailable for follow up and 7 stopped for a medical procedure. 320 participants with CF diagnosed clinically, by genotype or sweat test. Participants aged from 7 to 57 years, with FVC < 40 % predicted. Baseline lung function in the treatment group was lower than that of the control group, P < 0.05.
Interventions
Comparison of nebulized dornase alfa 2.5 mg od (n = 158) to placebo (n = 162) over 12 weeks.
Outcome measures
Measurements were taken on days 8, 15, 29, 57 and 85. Included in this review: mean change in % predicted FVC and FEV1, number of deaths and number experiencing adverse event, relative risk of one or more respiratory exacerbation.
Main 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 i.v. 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.
Authors' 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.