Recombinant human DNase I, or dornase alfa, is the first new therapy d
eveloped specifically for cystic fibrosis in almost 30 years. It selec
tively digests extracellular DNA and reduces the viscosity of purulent
sputum. In clinical trials dornase alfa modestly improved pulmonary f
unction, slightly decreasing the number of respiratory exacerbations r
equiring parenteral antibiotics compared with placebo. Phase III studi
es suggest that patients receiving dornase alfa also spend slightly fe
wer days in the hospital than those treated with placebo. The aerosoli
zed preparation is safe and generally well tolerated. Voice alteration
and sore throat are the most commonly reported adverse effects. Furth
er research is necessary to determine the optimum time to initiate the
rapy and to evaluate the agent's pharmacoeconomic impact on the treatm
ent of cystic fibrosis. Aerosolized dornase alfa should always be give
n in conjunction with standard cystic fibrosis therapies including ant
ibiotics, chest physiotherapy, and pancreatic enzyme supplementation.