DNase in stable cystic fibrosis infants: a pilot study.
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.


METHODS
open-label randomized placebo controlled cross-over pilot study.


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.
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.
Maartje Ten
MT
Berge
Els van der
Ev
Wiel
Harm A W M
HA
Tiddens
Peter J F M
PJ
Merkus
Wim C J
WC
Hop
Johan C
JC
de Jongste
D000280:Administration, Inhalation; D002675:Child, Preschool; D018592:Cross-Over Studies; D003550:Cystic Fibrosis / Q000175:diagnosis* / Q000188:drug therapy*; D003851:Deoxyribonucleases / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D009330:Nebulizers and Vaporizers; D010865:Pilot Projects; D010991:Plethysmography; D011336:Probability; D012016:Reference Values; D012129:Respiratory Function Tests; D018570:Risk Assessment; D012720:Severity of Illness Index; D016037:Single-Blind Method; D016896:Treatment Outcome
D003851:Deoxyribonucleases