DNase in stable cystic fibrosis infants: a pilot study

J Cyst Fibros. 2003 Dec;2(4):183-8. doi: 10.1016/S1569-1993(03)00090-0.


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.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial

MeSH terms

  • Administration, Inhalation
  • Child, Preschool
  • Cross-Over Studies
  • Cystic Fibrosis / diagnosis*
  • Cystic Fibrosis / drug therapy*
  • Deoxyribonucleases / therapeutic use*
  • Dose-Response Relationship, Drug
  • Drug Administration Schedule
  • Female
  • Follow-Up Studies
  • Humans
  • Infant
  • Male
  • Nebulizers and Vaporizers
  • Pilot Projects
  • Plethysmography
  • Probability
  • Reference Values
  • Respiratory Function Tests
  • Risk Assessment
  • Severity of Illness Index
  • Single-Blind Method
  • Treatment Outcome


  • Deoxyribonucleases