Analysis of mucolytic therapy use in cystic fibrosis based on national registries
https://doi.org/10.31146/2949-4664-apps-3-2-88-99
Abstract
An analysis of mucolytic drug use was conducted using data from national cystic fibrosis (CF) patient registries in Europe, the US, Canada, Australia, and Russia. Mucolytic therapy is a key component of CF treatment. Mucolytic drugs are used in patients of all ages. During the study period, dornase alfa was prescribed to the largest number of patients across all registries. In Russia, this drug is available to all patients, largely owing to the “14 High- Cost Nosologies” program and the presence of a dornase alfa biosimilar produced in Russia. According to registry data, dornase alfa was used more frequently in Russia than in other countries – up to 95.8% in 2019, with 25.9% of patients receiving a second dose intranasally (in 2023). In the US, dornase alfa was prescribed to up to 90% of patients, in Australia – up to 60%, in Canada – up to 52% of adult patients, and in the EU, more than 50% of patients received this drug. Among rapid-acting mucolytics, hypertonic saline inhalation was the most commonly prescribed, accounting for up to 77% of patients in the US, up to 72.7% in Russia, up to 52.3% in the EU, up to 45.4% in Australia, and up to 41.6% in Canada among adult CF patients. Mannitol inhalation was used in individual cases, primarily in adolescence (up to 15.7% of adolescents with CF in Australia in 2020). In recent years, a slight decrease in the use of mucolytic medications has been observed across all countries under study.
About the Authors
V. V. Shadrina
Research Clinical Institute of Childhood; Research Centre for Medical Genetics
Russian Federation
Russian Federation
Vera V. Shadrina, Cand. Sci. (Med.), Head of the Department of Hereditary and Metabolic Diseases; Leading Research Fellow of Cystic Fibrosis Department
24A, bldg. 1, Kominterna str., Mytishchi, 141009
1, Moskvorechye str., Moscow, 115522
E. I. Kondratyeva
Research Clinical Institute of Childhood; Research Centre for Medical Genetics
Russian Federation
Russian Federation
Elena I. Kondratyeva, Dr. Sci. (Med.), Prof., Deputy Director for Scientifi c Work, Head of Scientific and Clinical Department for Cystic Fibrosis; Head of Department for Medical Genetics of the Diseases of Respiratory System, Institute of Higher and Postgraduate Professional Education
Scopus ID: 35196167800
24A, bldg. 1, Kominterna str., Mytishchi, 141009
1, Moskvorechye str., Moscow, 115522
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For citations:
Shadrina V.V., Kondratyeva E.I. Analysis of mucolytic therapy use in cystic fibrosis based on national registries. Archives of Pediatrics and Pediatric Surgery. 2025;3(2):88-99. (In Russ.) https://doi.org/10.31146/2949-4664-apps-3-2-88-99
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