Dornase alfa, an inhaled drug, for treating lung disease in cystic fibrosis

Cystic fibrosis (CF) is a genetic disorder which affects the exocrine glands (sweat glands and others). People with CF have a greater risk of chronic lung disease, which can be life-threatening. Lung infections produce thick sputum (phlegm) which can block air passages. This can in turn cause further infection. Dornase alfa was developed to thin out this sputum. We included 15 trials in the review. Twelve trials compared dornase alfa to placebo or no dornase alfa treatment; one compared daily dornase alfa with hypertonic saline and alternate day dornase alfa; and two compared daily dornase alfa to hypertonic saline. The trials lasted from six days to two years. The review found evidence that dornase alfa improves lung function up to one month and this effect was also seen at six months in one trial. There were no serious adverse effects reported, only minor ones such as rash and change in voice. It's likely that further trials comparing daily dornase alfa with other regimens, e.g. alternate day dornase alfa or other mucolytics, will be important in the future.