Clinical and functional control of mucolytic therapy in children with cystic fibrosis

Abstract

The article describes the features of mucolytic therapy in patients with cystic fibrosis, as the leading pathogenetic mechanism of the disease lies in the compromised mucociliary transport due to the presence of viscous secretions. Particular attention is paid to the key mucolytic drug (endonuclear mucolytic) dornase alfa, which has three clinical effects: powerful mucolytic, anti-inflammatory and antibacterial. The extracellular DNA cleavage in secretions results in a decrease of bronchial mucus viscosity, and a decrease in elastase and IL-8 concentrations in sputum results in the reduction of lung tissue destruction in chronic bacterial inflammation. The authors pay attention to the adherence to the drug policy, possible dosing regimens and drug use variants to achieve a clinical effect. Also, the article extensively covers the results of international clinical studies on the use of dornase alfa in cystic fibrosis. The authors presented their own clinical observation – the experience of clinical and functional control over the efficacy of the drug when comparing two groups of children with cystic fibrosis: those who received (2018–2021) and those who did not receive dornase alfa (1995–1998 – before it was introduced into clinical practice). The remarkable thing is that the results of FEV1, MEF 50, MEF 75, PEF were significantly higher in the group of modern children with cystic fibrosis, who receive dornase alfa continuously as part of the backbone therapy, than in children of the same age 25 years ago. It has been substantiated that dornase alfa is one of the main components of the backbone therapy for patients with cystic fibrosis, which should be prescribed on an ongoing basis after the diagnosis has been established, under the functional control of its efficacy

Keywords

• cystic fibrosis
• dornase alfa
• mucolytic therapy
• mucostasis
• chronic bronchopulmonary process