Results of a prospective open observational study of the use of dornase alfa as part of complex therapy for patients with cystic fibrosis
V. V. Shadrina,
E. I. Kondratyeva,
A. Yu. Voronkova,
E. K. Zhekaite,
S. A. Krasovskiy,
E. L. Amelina,
A. V. Orlov,
Yu. S. Pinegina,
A. E. Lavrova,
S. V. Trishina,
D. I. Abdulganieva,
S. Yu. Semykin,
A. V. Vlasova https://doi.org/10.18093/0869-0189-2024-34-2-206-217
Abstract
The article discusses results of a prospective observational study of long-term use of the biosimilar dornase alfa (Tigerase) (Generium, Russia) as part of complex therapy in patients with cystic fibrosis in real clinical practice.
The aim was to analyze the outcomes of long-term use of the dornase alfa dornase alfa as part of complex therapy in patients with CF (protocol #DRN-CFR-N01).
Methods. The study included patients (n = 165) aged 5 years and older from 11 centers for treatment of cystic fibrosis in the Russian Federation with a confirmed diagnosis of cystic fibrosis who were prescribed dornase alfa by their attending physician.
Results. The analysis revealed that exacerbations of chronic pulmonary disease during the treatment with dornase alfa were observed in 29 (17.58%) patients included in the study. At the same time, there were no statistically significant changes in FEV1 and FVC (%) against baseline during the treatment in the study population. Adverse events related to the study drug were recorded in 9 (5.45%) patients.
Conclusion. Biosimilar dornase alfa demonstrated a favorable efficacy and safety profile in routine clinical practice, which confirms the results of previously published studies.
Supporting agencies: The observational trial was sponsored by JSC “Generium”
About the Authors
V. V. Shadrina
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation;
Federal State Budgetary Educational Institution of Higher Education “Perm State Medical University named after academician E.A.Wagner” of the Ministry of Health of the Russian Federation
Russian Federation
Russian Federation
Vera V. Shadrina, Candidate of Medicine, Leading Researcher, Scientific and Clinical Department of Cystic Fibrosis, Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation; Associate Professor, Department of Faculty and Hospital Pediatrics, Federal State Budgetary Educational Institution of Higher Education “Perm State Medical University named after academician E.A.Wagner” of the Ministry of Health of the Russian Federatio
ul. Moskvorechye 1, Moscow, 115522,
ul. Petropavlovskaya 26, Perm’, 614000
E. I. Kondratyeva
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation;
State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
Russian Federation
Russian Federation
Elena I. Kondratyeva, Doctor of Medicine, Professor, Head of the Scientific and Clinical Department of Cystic Fibrosis, Head of the Department of Genetics of Respiratory System Diseases, Institute of Higher and Additional Professional Education, Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation; Deputy Director for Research, State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
ul. Moskvorechye 1, Moscow, 115522,
ul. Bolshaya Serpukhovskaya 62, Moscow, 115093
A. Yu. Voronkova
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation;
State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
Russian Federation
Russian Federation
Anna Yu. Voronkova, Candidate of Medicine, Leading Researcher, Scientific and Clinical Department of Cystic Fibrosis, Federal State Budgetary Scientific
Institution “Research Centre for Medical Genetics”, Ministry of Science and
Higher Education of the Russian Federation; Pediatrician, Department of Cystic Fibrosis, State Budgetary Healthcare Institution of the Moscow region
“Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow
Region
ul. Moskvorechye 1, Moscow, 115522,
ul. Bolshaya Serpukhovskaya 62, Moscow, 115093
E. K. Zhekaite
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation;
State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
Russian Federation
Russian Federation
Elena K. Zhekaite, Candidate of Medicine, Senior Researcher, Department of Cystic Fibrosis, Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation; Pediatrician, Department of Cystic Fibrosis, State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
ul. Moskvorechye 1, Moscow, 115522,
ul. Bolshaya Serpukhovskaya 62, Moscow, 115093
S. A. Krasovskiy
Federal State Budgetary Institution “Pulmonology Scientific Research Institute” under Federal Medical and Biological Agency of Russian Federation
Russian Federation
Russian Federation
Stanislav А. Krasovskiy, Candidate of Medicine, Senior Researcher, Laboratory of Cystic Fibrosis
Orekhovyy bul’var 28, Moscow, 115682
Author ID: 688178
E. L. Amelina
Federal State Budgetary Institution “Pulmonology Scientific Research Institute” under Federal Medical and Biological Agency of Russian Federation
Russian Federation
Russian Federation
Elena L. Amelina, Candidate of Medicine, Head of the Cystic Fibrosis Department
Orekhovyy bul’var 28, Moscow, 115682
A. V. Orlov
St. Petersburg State Budgetary Institution of Health Care “Children’s City Hospital of St. Olga”;
Federal State Budgetary Educational Institution of Higher Education “North-Western State Medical University named after I.I.Mechnikov”, Ministry of Health of the Russian Federation
Russian Federation
Russian Federation
Aleksander V. Orlov, Candidate of Medicine, Head of the Infectious-Boxing Department, St. Petersburg State Budgetary Institution of Health Care “Children’s City Hospital of St. Olga”; Associate Professor, Department of Pediatrics and Neonatology, Federal State Budgetary Educational Institution of Higher Education “North-Western State Medical University named after I.I.Mechnikov”, Ministry of Health of the Russian Federation
ul. Zemledel’cheskaya 2, Saint-Petersburg, 194156,
ul. Kirochnaya 41, Saint-Petersburg, 191015
Yu. S. Pinegina
State Budgetary Healthcare Institution of the Novosibirsk region “State Novosibirsk Regional Clinical Hospital”
Russian Federation
Russian Federation
Yuliya S. Pinegina, Сandidate of Medicine, Chief Pediatric Pulmonologist of
the Novosibirsk region, Head of the Pediatric Pulmonology Department
ul. Nemirovichа-Danchenko 130, Novosibirsk, 630087
A. E. Lavrova
Federal State Budgetary Educational Institution of Higher Education “Privolzhsky Research Medical University” of the Ministry of Health of the Russian Federation
Russian Federation
Russian Federation
Alla E. Lavrova, Doctor of Medicine, Chief Researcher, Department of the Clinic
of Gastroenterology and Metabolic Disorders, Institute of Pediatrics, University
Hospital
ul. Semashko 22, Nizhny Novgorod, 603950
S. V. Trishina
Medical Academy named after S.I.Georgievskiy is the structural unit of the Federal State Autonomous Educational Institution of Higher Education “Crimean Federal University named after V.I.Vernadskiy” of the Ministry of Science and Higher Education of the Russian Federation
Russian Federation
Russian Federation
Svetlana V. Trishina, Doctor of Medicine, Professor, Chief Freelance Specialist, Ministry of Health of the Republic of Crimea, Head of the Department of Propaedeutics of Pediatrics, Medical Academy named after S.I.Georgievskiy is the structural unit
bul’v. Lenina 5/7, Simferopol’, Crimea Republic, 295051
D. I. Abdulganieva
Federal State Budgetary Educational Institution of Higher Education “Kazan’ State Medical University” of the Ministry of Health of the Russian Federation
Russian Federation
Russian Federation
Diana I. Abdulganieva, Doctor of Medicine, Professor, Head of the Department Hospital Therapy
ul. Butlerova 49, Kazan’, Tatarstan Republic, 420012
S. Yu. Semykin
Separate Structural Unit “Russian Children’s Clinical Hospital” of the Federal State Autonomous Educational Institution of Higher Education “N.I.Pirogov Russian National Research Medical University” of the Ministry of Health of the Russian Federation
Russian Federation
Russian Federation
Sergey Yu. Semykin, Сandidate of Medicine, Head of the Pediatric Department
Leninskiy prospekt, 117, Moscow, 119571
A. V. Vlasova
State Budgetary Healthcare Institution “Morozov Children’s City Clinical Hospital of the Moscow Health Department”
Russian Federation
Russian Federation
Anna V. Vlasova, Сandidate of Medicine, Clinical Pharmacologist, Head of
the Department of Clinical Pharmacology
4th Dobryninskiy per. 1/9, Moscow, 19049
References
1. Krasovskiy S.A., Starinova M.A., Voronkova A.Yu. et al., eds. [Register of patients with cystic fibrosis in the Russian Federation. 2021]. St. Petersburg: Blagotvoritel’nyy fond “Ostrova”; 2023. Available at: https://mukoviscidoz.org/doc/registr/registr_systicfibrosis_brochure_19_10.pdf (in Russian).
2. Sherman V.D., Kapranov N.I., Kashirskaya N.Yu [Dornase alpha (Pulmozyme) for the complex treatment of bronchopulmonary process in cystic fibrosis patients]. Farmateka. 2011; 11 (224): 42– 45. Available at: https://journals.eco-vector.com/2073-4034/article/view/278929 (in Russian).
3. Aksenova M.S., Bocharova E.N., Abbasova S.G. et al. [Comparability of the reference drug Pulmozim and a similar drug Tigerase in terms of their pharmacodynamic, toxicological and pharmacokinetic properties]. Biomeditsina. 2023; 19 (1): 47–60. DOI: 10.33647/2074-5982-19-1-47-60 (in Russian).
4. Amelina E.L., Krasovskiy S.A., Abdulganieva D.I. et al. [Efficacy and safety of the biosimilar medicinal product Tigerase® (dornase alfa) in long-term symptomatic treatment of patients with cystic fibrosis: results of a phase III clinical trial]. Pul’monologiya. 2019; 29 (6): 695–706. DOI: 10.18093/0869-0189-2019-29-6-695-706 (in Russian).
5. Ministry of Health of the Russian Federation. [Clinical guidelines: Cystic fibrosis in children and adults. 2021]. Available at: https://cr.minzdrav.gov.ru/recomend/372_2 [Accessed: October 02, 2024] (in Russian).
6. Chuchalin A.G., Aisanov Z.R., Chikina S.Yu. et al. [Federal guidelines of Russian Respiratory Society on spirometry]. Pul’monologiya. 2014; (6): 11–24. DOI: 10.18093/0869-0189-2014-0-6-11-24 (in Russian).
7. Miller M.R., Hankinson J., Brusasco V. et al. Standardisation of spirometry. Eur. Respir. J. 2005; 26 (2): 319–338. DOI: 10.1183/09031936.05.00034805.
8. Kondratyeva E.I., Voronkova A.Yu., Kashirskaya N.Yu. et al. [Russian registry of patients with cystic fibrosis: lessons and perspectives]. Pul’monologiya. 2023; 33 (2): 171–181. DOI: 10.18093/0869-0189-2023-33-2-171-181 (in Russian).
9. Kerem E., Viviani L., Zolin A. et al. Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS patient registry. Eur. Respir. J. 2014; 43 (1): 125–133. DOI: 10.1183/09031936.00166412.
10. Kondratyeva E.I., Shadrina V.V., Furman E.G. et al. [Estimation of possibility of application of Tigerase® (Dornase alfa) drug on the results of a multicenter scientific program of post marketing use of the drug]. Pediatriya im. G.N.Speranskogo. 2021; 100 (3): 218–226. DOI: 10.24110/0031-403X-2021-100-3-218-226 (in Russian).
11. Kashirskaya N.Yu., Kondratyeva E.I., Krasovskiy S.A. et al. [Register of patients with cystic fibrosis in the Russian Federation. 2019]. Moscow: Medpraktika-M; 2021. Available at: https://mukoviscidoz.org/doc/registr/site_Registre_2019.pdf (in Russian).
Supplementary files
Review
For citations:
Shadrina V.V., Kondratyeva E.I., Voronkova A.Yu., Zhekaite E.K., Krasovskiy S.A., Amelina E.L., Orlov A.V., Pinegina Yu.S., Lavrova A.E., Trishina S.V., Abdulganieva D.I., Semykin S.Yu., Vlasova A.V. Results of a prospective open observational study of the use of dornase alfa as part of complex therapy for patients with cystic fibrosis. PULMONOLOGIYA. 2024;34(2):206-217. (In Russ.) https://doi.org/10.18093/0869-0189-2024-34-2-206-217
Views:
348
Email this article 