Dornase alfa ( Pulmozyme ) has been shown to reduce markers of inflammation and neutrophil-associated metalloproteinases in cystic fibrosis, suggesting a potential benefit from use of this therapy early in the disease.
However, observational studies indicate that Dornase alfa is often reserved for sicker patients.
A 2-year, early intervention study of Dornase alfa in cystic fibrosis patients with early lung disease demonstrated significant improvements in lung function and risk of exacerbation compared to placebo.
A more recent analysis, using the database of the large observational Epidemiologic Study of Cystic Fibrosis ( ESCF ), found that initiation of Dornase alfa has the potential to alter the course of cystic fibrosis by decreasing the rate of lung function decline in children and adults.
These encouraging results, possibly linked to indirect effects on inflammation, suggest a greater role for Dornase alfa therapy in the early treatment of cystic fibrosis, where it may help preserve lung function and potentially extend survival. ( Xagena )
Konstan MW, Ratjen F, J Cyst Fibros 2012;11:78-83