AEROSOL DELIVERY AND SAFETY OF RECOMBINANT HUMAN DEOXRYRIBONUCLEASE IN YOUNG-CHILDREN WITH CYSTIC-FIBROSIS - A BRONCHOSCOPIC STUDY

The purpose of this study was to assess the delivery to the lungs and the short-term safety of recombinant human deoxyribonuclease (rhDNase, Pulmozyme) in children with cystic fibrosis younger than 5 years of a ge compared with elder children. Patients between the ages of 3 months and 10 years had bronchoscopic examination with bronchoalveolar lavag e (BAL) after administration of an aerosol dose of 2.5 mg of rhDNase. After recovery from the procedure, patients were discharged home for a n additional 13 days of rhDNase therapy. During this time adverse even ts were recorded to assess short-term safety. A total of 98 patients w ere enrolled, 65 (66%) aged 3 months to 5 years and 33 (34%) aged 5 Se ars to 10 years. Deoxyribonuclease concentrations in BAL fluid were va riable (interquartile range, 752 to 3943 mu g/mL epithelial lining flu id [ELF]) and did not depend on patient age, weight, or height or diff er when delivered through a mouthpiece or mask. The median value for t he BAL DNA concentration in the younger group was 432 mu g/mL ELF comp ared with 703 mu g/mL ELF in the older patients. This study demonstrat es the value of bronchoscopy and BAL for assessing nebulized medicatio n delivery in young children and shows that aerosolized medications ca n be delivered to and are present in comparable amounts in the lower a irways of younger and older children. Exposure to rhDNase appears to b e safe over 2 weeks in infants and young children with cystic fibrosis .