Efficacy of human recombinant DNase in pediatric patients with cystic fibrosis

Abstract

Background: Most respiratory complications in cystic fibrosis (CF) arise from abnormally viscid mucus, and rhDNase has shown to be effective in enhancing mucous clearance. We explored the responses to rhDNase in a Mexican population of CF patients.

Methods: Patients with CF received aerosolized rhDNase (2.5 mg daily) during 3 months, followed by daily aerosolized placebo during 3 months.

Results: A total of 21 CF patients entered the study (11.1 +/- 0.5 years of age, mean +/- SEM, 10 girls): 15 patients (71%) had basal forced vital capacity (FVC) higher than the 70% predicted value, and the remainder of the patients had an FVC of between 30 and 70%. As a group, rhDNase progressively increased the forced expiratory flow at 1 sec (FEV1) as well as the FVC, reaching statistical significance (p <0.005) at the end of the third month of treatment. Sputum production and difficulty to expectorate or to breathe also improved during the rhDNase treatment period (p <0.05 to p <0.001). All these changes progressively decreased to basal values after 3 months with aerosolized placebo. Adverse reactions were almost null, with a sole patient reporting dysphonia.

Conclusions: Aerosolized rhDNase was effective in progressively improving respiratory function and symptoms in most CF patients.