How effective is Alfa deoxyribonuclease inhaler (Pulmozyme) in treating cystic fibrosis

A randomized, double-blind, placebo-controlled study to determine the effect of once-daily and twice-daily dosing of Pulmozyme on exacerbation of respiratory symptoms requiring parenteral antibiotics and lung function. A total of 968 adults and children with cystic fibrosis received treatment as outpatients for 24 weeks.

Results: One or more exacerbations occurred in 27% of patients taking placebo, 22% of patients taking Pulmozyme once daily, and 19% of patients taking Pulmozyme twice daily. Compared with placebo, once-daily and twice-daily administration of Pulmozyme reduced the age-adjusted risk of respiratory exacerbation by 28% (P=0.04) and 37% (P<0.01), respectively.

During the study period, once-daily and twice-daily administration of Pulmozyme improved forced expiratory volume in 1 second by a mean (+/-SD) of 5.8+/-0.7 and 5.6+/-0.7%, respectively. All patients had no allergic reactions. Voice changes and laryngitis were more common in Pulmozyme-treated patients than in those who received placebo, but were rarely severe and resolved within 21 days of onset.

Conclusions: In patients with cystic fibrosis, administration of Pulmozyme reduced but did not eliminate exacerbations of respiratory symptoms, modestly improved lung function, and was well tolerated.

There is evidence that inhaled (Pulmozyme) treatment improves lung function in people with cystic fibrosis compared with placebo in trials lasting between one month and two years. Lung exacerbations were reduced in trials lasting six months or longer. Voice changes and rash appear to be the only adverse events reported with increased frequency in randomized controlled trials. There is insufficient evidence to determine whether alfa deoxyribonuclease inhaler (Pulmozyme) is better than other hyperosmolar drugs in improving lung function.

One trial (23 children) showed greater improvement in forced expiratory volume in one second with alfa deoxyribonuclease inhaler (Pulmozyme) compared with hypertonic saline (low-certainty evidence).

Pulmozyme improves lung function in people with cystic fibrosis by reducing lung secretions and reducing the risk of respiratory infections. Pulmozyme is a synthetic protein that breaks down excess DNA in the lung secretions of people with cystic fibrosis. Pulmozyme is recommended for inhalation once daily in a 2.5 mg disposable ampoule using a recommended nebulizer.

Common adverse reactions to Pulmozyme include voice changes, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, FVC decrease ≥10%, fever, and dyspnea. It is recommended that patients take medication under the guidance of a doctor and receive symptomatic treatment.

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References

[1.]Fuchs HJ, Borowitz DS, Christiansen DH, Morris EM, Nash ML, Ramsey BW, Rosenstein BJ, Smith AL, Wohl ME. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med. 1994 Sep 8;331(10):637-42. doi: 10.1056/NEJM199409083311003. PMID: 7503821.

[2.]Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2018 Sep 6;9(9):CD001127. doi: 10.1002/14651858.CD001127.pub4. Update in: Cochrane Database Syst Rev. 2021 Mar 18;3:CD001127. PMID: 30187450; PMCID: PMC6513278.