Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients.
BACKGROUND
Dornase alfa administered via jet nebulizer is indicated as a chronic respiratory medication for cystic fibrosis (CF) patients. Efficacy and safety of dornase alfa via an electronic nebulizer with vibrating membrane technology have not been formally assessed in randomized clinical trials.


METHODS
87 CF patients (≥6 years) were randomized in a crossover study to receive dornase alfa 2.5 mg/d in 2-week periods with the Pari eRapid and Pari LC Plus jet nebulizers. The primary end point was comparison of forced expiratory volume in the first second. Safety, quality of life, and treatment satisfaction/preference were also compared between devices.


RESULTS
Lung function was equivalent between nebulizers. Most domain scores from the Cystic Fibrosis Questionnaire-Revised and Treatment Satisfaction Questionnaire for Medication instruments were similar but patients strongly preferred the eRapid. Mean patient-reported administration times were shorter with the eRapid vs the LC Plus (2.7 vs 10.2 min). Adverse events were similar between devices.


CONCLUSIONS
Administration of dornase alfa via the eRapid nebulizer resulted in comparable efficacy and safety, shorter nebulization times, and higher patient preference.
Boston Children's Hospital, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA. Electronic address: gregory.sawicki@childrens.harvard.edu.
Gregory S
GS
Sawicki
1 DNA Way, Genentech, Inc., South San Francisco, CA 94080, USA.
Will
W
Chou
1 DNA Way, Genentech, Inc., South San Francisco, CA 94080, USA.
Karina
K
Raimundo
1 DNA Way, Genentech, Inc., South San Francisco, CA 94080, USA.
Ben
B
Trzaskoma
Rainbow Babies and Children's Hospital, Case Western Reserve University School of Medicine, 11100 Euclid Avenue, Cleveland, OH 44106, USA.
Michael W
MW
Konstan
D000293:Adolescent; D002648:Child; D003550:Cystic Fibrosis / Q000188:drug therapy*; D003850:Deoxyribonuclease I / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006801:Humans; D008297:Male; D009330:Nebulizers and Vaporizers; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects; D016896:Treatment Outcome
D011994:Recombinant Proteins; D003850:Deoxyribonuclease I; C568813:dornase alfa
Cystic fibrosis; Dornase alfa; Nebulizers