Dornase alfa (brand name Pulmozyme) is a synthetic protein that breaks down excess DNA in the pulmonary secretions of people with cystic fibrosis. It improves lung function in CF by thinning thick mucus secretions, allowing the air to flow better, preventing bacteria from causing infections.1
Dornase alfa was approved for use by the FDA in 1993 after a Phase 3 study demonstrated improvement in lung function and reduction in pulmonary exacerbations.2 The solution is inhaled and usually taken once or twice a day.3 It should only be used with a recommended nebulizer after learning the proper technique. Dornase alfa comes as a single-use ampule that should be stored under refrigeration and away from strong light. Then an approved nebulizer, with its components (tubing and mouthpiece) are essential for the correct treatment to be done.
Dornase alfa will treat CF but won’t cure it, so it should be used even if there are no symptoms.
Some of the side effects people may experience are voice changes, sore throat, hoarseness, eye irritation, and rash.1
Dornase alfa is safe and effective in children over 5 years old. Nevertheless, for younger children, its use should be considered when there may be a potential benefit in lung function or who may be at risk of respiratory tract infection. The safety of daily inhalation of dornase alfa has been studied in 98 children (65 of them aged 3 months to <5 years and 33 aged 5 to <10 years). The side effects observed were similar to those seen in older patients.
There are no generics available for dornase alfa.
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