Study design (if review, criteria of inclusion for studies)
open-label randomized placebo controlled cross-over pilot study.
Participants
9 CF patients (0.7-1.9 years)
Interventions
nebulised rhDNase (2.5 mg) and NaCl 0.9% (10 ml) via jet nebulizer once daily during 2-week treatment blocks.
Outcome measures
Measurements were performed at baseline and after treatment blocks and consisted of lung function tests (plethysmography and tidal rapid thoraco-abdominal compression technique), overnight pulse oximetry, and daily symptom scores.
Main results
DNase treatment and the different assessments were well tolerated by all children and their parents. Lung function showed increased airway patency after treatment with rhDNase (P < 0.001), but not after NaCl 0.9%. Overnight pulse oximetry and daily symptom scores did not change during the study period.
Authors' conclusions
This pilot study indicates that objective assessment of the effects of rhDNase is feasible in infants with CF who have little or no respiratory symptoms. Our results warrant a larger randomized placebo-controlled trial.
Keywords: Child; Deoxyribonuclease; Drug Administration Schedule; Airway clearance drugs -expectorants- mucolytic- mucociliary-; Infant; Inhalation OR nebulised; nebuliser; non pharmacological intervention - devices OR physiotherapy; pharmacological_intervention; Respiratory System Agents; Dornase alpha; Pulmozyme;