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Updated: Aug 21, 2026
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Published on: April 11, 2018
DNase in stable cystic fibrosis infants: a pilot study
Maartje Ten Berge1, Els van der Wiel, Harm A W M Tiddens
1Department of Pediatrics, division of Respiratory Medicine, Erasmus MC--University Medical Center Rotterdam/Sophia Children's Hospital, P.O. Box 2060, 3000, CB, Rotterdam, Netherlands.
Objective:
To assess the feasibility of measuring short-term effects of inhaled recombinant human deoxyribonuclease (rhDNase, Pulmozyme on lung function, pulse oximetry and symptom scores in infants and toddlers with stable cystic fibrosis.
Design:
open-label randomized placebo controlled cross-over pilot study.
Patients And Methods:
We treated nine CF patients (0.7-1.9 years) with nebulised rhDNase (2.5 mg) and NaCl 0.9% (10 ml) via jet nebulizer cross-over once daily during 2-week treatment blocks. 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.
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.
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.
