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RECOMBINANT DNase HAS MODEST BENEFITS IN CYSTIC FIBROSIS.

Preliminary studies suggested that recombinant human DNase (rhDNase) improves pulmonary function in patients with cystic fibrosis by reducing the viscosity of respiratory secretions. This randomized, double-blind trial, conducted at 51 institutions, tested aerosolized rhDNase in 968 children and adults with cystic fibrosis.

The patients received a 24-week course of rhDNase (2.5 mg, either once or twice daily) or placebo in addition to conventional care. rhDNase significantly reduced the proportion of patients with exacerbations of respiratory symptoms that necessitated parenteral antibiotics (22 percent with once-daily treatment and 19 percent in the twice-daily group, versus 27 percent in the placebo group). In addition, as compared with placebo, rhDNase therapy led to a 6 percent improvement in FEV1, fewer days of hospitalization, and a reduction in reported symptoms. All these differences were statistically significant. rhDNase was generally well tolerated.

Comment: The clinical benefits of rhDNase were modest, and were demonstrable in this study because of its large size. As an editorial notes, the next task is to determine which patients are most likely to benefit from this therapy, given its high cost (currently about $27 per dose).

— AS Brett

Published in Journal Watch General Medicine September 20, 1994

Citation(s):

Davis PB. Evolution of therapy for cystic fibrosis. N Engl J Med 1994 Sep 8 331 672-673.

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• Medline abstract (Free)

Fuchs HJ et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med 1994 Sep 8 331 637-642.

• Original article (Subscription may be required)
• Medline abstract (Free)