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.

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doi.org/10.1016/S1569-1993(03)00090-0, hdl.handle.net/1765/68463
Journal of Cystic Fibrosis
Erasmus MC: University Medical Center Rotterdam

ten Berge, M, van der Wiel, E.C, Tiddens, H.A.W.M, Merkus, P.J.F.M, Hop, W.C.J, & de Jongste, J.C. (2003). DNase in stable cystic fibrosis infants: A pilot study. Journal of Cystic Fibrosis, 2(4), 183–188. doi:10.1016/S1569-1993(03)00090-0