pulmoПульмонологияPULMONOLOGIYA0869-01892541-9617Scientific and Practical Journal “PULMONOLOGIYA” LLC10.18093/0869-0189-2025-35-2-167-176pulmo-4714Research ArticleПЕРЕДОВАЯ СТАТЬЯEDITORIALНовые возможности таргетной терапии муковисцидозаNew possibilities for targeted therapy of cystic fibrosishttps://orcid.org/0000-0001-6395-0407КондратьеваЕ. И.KondratyevaE. I.

Елена Ивановна Кондратьева, д. м. н., профессор, заместитель директора Центра, руководитель отдела, заведующая кафедрой

Центр муковисцидоза; научно-клинический отдел муковисцидоза; Институт высшего и дополнительного профессионального образования; кафедра генетики болезней дыхательной системы

115522; ул. Москворечье, 1; Москва; 141009;  ул. Коминтерна, 24А, стр. 1; Московская обл;, Мытищи

тел.: (495) 324-20-24

Scopus ID: 35196167800; Web of Science Researcher ID: АВВ-9783–2021

Elena I. Kondratyeva, Doctor of Medicine, Professor, Deputy Director of the Center, Head of the Department

Cystic Fibrosis Center; Scientific and Clinical Department of Cystic Fibrosis; Department of Genetics of Diseases of the Respiratory System

115522; ul. Moskvorechye 1; Moscow; 141009; ul. Kominterna 124A, build. 1; Moscow Region; Mytishchi

tel.: (495) 324-20-24

Scopus ID: 35196167800; Web of Science Researcher ID: АВВ-9783–2021

elenafpk@mail.ruhttps://orcid.org/0000-0002-5999-2150АвдеевС. Н.AvdeevS. N.

Сергей Николаевич Авдеев, д. м. н., профессор, академик Российской академии наук; директор центра, заведующий кафедрой, руководитель отдела, главный внештатный пульмонолог Министерства здравоохранения Российской Федерации

Национальный медицинский исследовательский центр по профилю «Пульмонология»; Институт клинической медицины имени Н.В.Склифосовского; кафедра пульмонологии; Федеральное государственное бюджетное учреждение «Научно-исследовательскийинститут пульмонологии» Федерального медико-биологического агентства; клинический отдел

119991; ул. Трубецкая, 8, стр. 2; Москва

тел.: (495) 708-35-76

Sergey N. Avdeev, Doctor of Medicine, Professor, Academician of Russian Academy of Sciences, Director of the National Medical Research Center, Head of the Department, Chief Pulmonologist of the Ministry of Health of the Russian Federation

National Medical Research Center for Pulmonology; N.V. Sklifosovsky Institute of Clinical Medicine; Department of Pulmonology; Federal State Budgetary Institution “Pulmonology Scientific Research Institute” under Federal Medical and Biological Agency of Russian Federation; Clinical Department

119991; ul. Trubetskaya 8, build. 2; Moscow

tel.: (495) 708-35-76

serg_avdeev@list.ruhttps://orcid.org/0000-0002-3133-8018КуцевС. И.KutsevS. I.

Сергей Иванович Куцев, д. м. н., академик Российской академии наук, директор ФГБУ, главный внештатный специалист по медицинской генетике Министерства здравоохранения Российской Федерации, председательЭтического комитета Министерства здравоохранения Российской Федерации; президент Ассоциации медицинских генетиков

115522; ул. Москворечье, 1; Москва

тел: (495) 612-00-37

Sergey I. Kutsev, Doctor of Medicine, Professor, Academician of Russian Academy of Sciences, Director of the Federal State Budgetary Scientific Institution, Chief Freelance Specialist in Medical Genetics, Ministry of Health of the Russian Federation, Chairman of the Ethics Committee, Ministry of Health of the Russian Federation, President of the Association of Medical Geneticists

115522; ul. Moskvorechye 1; Moscow

tel: (495) 612-00-37

mgnc@med-gen.ruФедеральное государственное бюджетное научное учреждение «Медико-генетический научный центр имени академика Н.П.Бочкова» Министерства науки и высшего образования Российской Федерации; Государственное бюджетное учреждение здравоохранения Московской области «Научно-исследовательский клинический институт детства Министерства здравоохранения Московской области»РоссияFederal 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 RegionRussian FederationФедеральное государственное автономное образовательное учреждение высшего образования Первый Московский государственный медицинский университет имени И.М.Сеченова Министерства здравоохранения Российской Федерации (Сеченовский Университет)РоссияFederal State Autonomous Educational Institution of Higher Education I.M.Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation (Sechenov University)Russian FederationФедеральное государственное бюджетное научное учреждение «Медико-генетический научный центр имени академика Н.П.Бочкова» Министерства науки и высшего образования Российской ФедерацииРоссияFederal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian FederationRussian Federation202518042025352167176Copyright © Кондратьева Е.И., Авдеев С.Н., Куцев С.И., 20252025Кондратьева Е.И., Авдеев С.Н., Куцев С.И.Kondratyeva E.I., Avdeev S.N., Kutsev S.I.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://journal.pulmonology.ru/pulm/article/view/4714

   Муковисцидоз (МВ) – это аутосомно-рецессивное заболевание, вызванное дисфункцией белка трансмембранного регулятора проводимости МВ (CFTR), которое может манифестировать с рождения и прогрессирует на протяжении всей жизни человека. МВ приводит к мультисистемному поражению, при котором значительно сокращается продолжительность жизни.

   Целью обзора являлось проведение анализа применения CFTR-модулятора элексакафтор (ELX) / тезакафтор (TEZ) / ивакафтор (IVA) для обоснования применения модулятора нового поколения ванзакафтор (VNZ) / TEZ / дейтивакафтор (D-IVA) и оценки его эффективности.

   Результаты

   Результаты. По результатам анализа 35 научных публикаций о таргетной терапии МВ и по данным исследований III фазы применения комбинации VNZ / TEZ / D-IVA описаны эффективность и безопасность таргетной терапии МВ.

   Заключение

   Заключение. Отмечено, что VNZ / TEZ / D-IVA – это следующее поколение CFTR-модуляторов, обладающее повышенными возможностями для дальнейшего улучшения клинических результатов и еще большего увеличения долгосрочных результатов за счет восстановления нормальной функции CFTR-канала у большего числа пациентов с МВ, предлагающее большие удобства для больных и лиц, осуществляющих уход.

   Cystic fibrosis (CF) is an autosomal recessive disorder caused by dysfunction of the CF transmembrane conductance regulator (CFTR) protein that can manifest at birth and progresses throughout life. CF results in multisystem disease with significantly reduced life expectancy.

   The aim of the review was to analyze the use of the CFTR modulator elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) to justify the use of the new-generation modulator vanzacaftor (VNZ) / TEZ / deitivacaftor (D-IVA) and evaluate its efficacy.

   Results

   Results. We analyzed 35 scientific publications on CF and its targeted therapy and described the efficacy and safety of the VNZ/TEZ/D-IVA combination in patients with CF (n = 1,049) according to the phase III studies.

   Conclusion

   Conclusion. VNZ/TEZ/D-IVA is a next generation CFTR modulator with increased potential to further improve clinical outcomes and further enhance long-term outcomes by restoring normal CFTR function in a larger number of CF patients. Additionally, VNZ/TEZ/D-IVA may improve treatment convenience for patients and caregivers.

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The authors declare that there are no conflicts of interest present.