Pharmacology, clinical efficacy and safety of recombinant human DNase in cystic fibrosis

Abstract

Recombinant human DNase (rhDNase) is a mucolytic agent that is primarily used to improve mucociliary clearance in cystic fibrosis (CF). rhDNase is a recombinant human enzyme that is synthesized in a Chinese hamster ovary cell line. rhDNase enzymatically cleaves extracellular DNA into molecules of shorter length. CF sputum shows high concentrations of DNA released by disintegrating inflammatory cells. Free DNA contributes to the abnormally high viscosity of CF sputum and therefore forms an important target in the treatment of CF lung disease. Clinical studies have shown that daily nebulization of rhDNase is associated with an increase in lung function and a decrease in the frequency of exacerbations in patients with CF.

Keywords::

• chemistry
• cystic fibrosis
• dornase-α
• efficacy
• Pulmozyme^®
• rhDNase
• safety

Financial & competing interests disclosure

H Tiddens has received, within the last 3 years, honoraria and travel expenses for lectures from Hoffman-La-Roche. He holds no stock options. The BV Kindergeneeskunde of the Erasmus MC – Sophia Children’s Hospital has, in the last 3 years, received unconditional research grants from Hoffman-La-Roche Netherlands BV. E Bakker is a research fellow who’s salary has been payed by an unconditional research grant from Hoffman-La-Roche Netherlands BV. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance outside the ErasmusMC was used in the preparation of the manuscript. We would like to thank Ko Hagoort for critical reading of the manuscript.