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Evidence summaries

Dornase Alfa for Cystic Fibrosis

Dornase alfa appears to decrease pulmonary exacerbations and improve lung function compared with placebo in cystic fibrosis. Level of evidence: "B"

A Cochrane review [Abstract] 1 included 19 trials with a total of 2 565 patients. 15 studies compared dornase alfa to placebo or no dornase alfa treatment; one compared daily dornase alfa with hypertonic saline and alternate day dornase alfa; two compared daily dornase alfa to hypertonic saline; and one compared dornase alfa to mannitol and the combination of both drugs. Study duration varied from 6 days to 3 years. The number of deaths was not significant between treatment groups. Compared to placebo, forced expiratory volume at one second improved in the intervention groups, with significant differences at one month, three months, six months and two years. There was also a significant improvement in lung clearance index at one month and a decrease in pulmonary exacerbations in trials of longer duration. There was no excess of adverse effects except voice alteration and rash.

Dornase alfa compared with placebo or no dornase alfa treatment for cystic fibrosis

OutcomesRelative effect(95% CI)Assumed risk - Control - Placebo or no dornase alfaCorresponding risk - Intervention - Dornase alfa (95% CI)No of Participants(studies) Quality of evidence
Relative mean percentage change in FEV1 (% predicted)at 3 mo-The relative mean change was 2.1The relative mean change was 7.3 higher(4.04 higher to 10.56 higher)320 (1) Moderate
Relative mean percentage change in FEV1 (% predicted)at 6 mo-The relative mean percentage change was 0The relative mean percentage change was 5.8 higher(3.99 higher to 7.61 higher)647 (1) High
Number of people experiencing exacerbationsat up to 2 years0.78 (0.62 to 0.96)252 per 1000196 per 1000(156 to 242)1157(3) Moderate

Comment: The quality of evidence is downgraded by study quality (unclear allocation concealment).

    References

    • Yang C, Chilvers M, Montgomery M et al. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev 2016;(4):CD001127. [PubMed].

Primary/Secondary Keywords