rhDNase in cystic fibrosis

Cystic fibrosis (CF) affects approximately 1 in 2500 live births in the UK Caucasian population. Morbidity and mortality closely reflect the degree of pulmonary involvement. rhDNase is a new form of therapy in CF care. Phase 1, 2 and 3 trials have shown it to be well-tolerated, with patients showing initial increases in respiratory function of around 13% from baseline. A years prescription costs $7500. This review discusses our views on how this expensive new therapy can best be used.