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J Cyst Fibros. 2003 Dec;2(4):183-8.

DNase in stable cystic fibrosis infants: a pilot study.

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1
Department of Pediatrics, division of Respiratory Medicine, Erasmus MC--University Medical Center Rotterdam/Sophia Children's Hospital, P.O. Box 2060, 3000, CB, Rotterdam, Netherlands.

Abstract

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.

PMID:
15463871
DOI:
10.1016/S1569-1993(03)00090-0
[Indexed for MEDLINE]
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