pulmoПульмонологияPULMONOLOGIYA0869-01892541-9617Scientific and Practical Journal “PULMONOLOGIYA” LLC10.18093/0869-0189-2025-35-6-875-882pulmo-4824Research ArticleОБЗОРЫREVIEWНовый трехкомпонентный CFTR-модулятор ванзакафтор / тезакафтор / дейтивакафтор (Алифтрек®): место в лечении муковисцидозаThe new triple CFTR modulator vanzacaftor/tezacaftor/ deutivacaftor (Aliftrek®) and its place in the treatment of cystic fibrosishttps://orcid.org/0000-0003-0503-6371КаширскаяН. Ю.KashirskayaN. Yu.

Каширская Наталия Юрьевна – д. м. н., профессор, главный научный сотрудник лаборатории генетической эпидемиологии ; профессор кафедры педиатрии

Scopus ID: 6507308033; WoS Researcher ID: C-6404-2012; РИНЦ ID: 93625

115522, Москва, ул. Москворечье, 1, тел.: (499) 324-12-24 

129110, Москва, ул. Щепкина, 61 / 2, корп. 1 

Nataliya Yu. Kashirskaya, Doctor of Medicine, Professor, Chief Researcher, Laboratory of Genetic Epidemiology; Professor, Department of Pediatrics

ul. Moskvorechye 1, Moscow, 115522, tel.: (499) 324-12-24

ul. Shchepkina 61/2, Moscow, 129110 

kashirskayanj@mail.ruhttps://orcid.org/0000-0002-5356-9415АмелинаЕ. Л.AmelinaE. L.

Амелина Елена Львовна – к. м. н., заведующая лабораторией муковисцидоза

115682, Москва, Ореховый бульвар, 28, тел.: (926) 205-03-91 

Elena L. Amelina, Candidate of Medicine, Head of the Cystic Fibrosis Laboratory 

Orekhovyy bul’var 28, Moscow, 115682, tel.: (926) 205-03-91 

eamelina@mail.ruhttps://orcid.org/0000-0003-3586-3458ЗинченкоР. А.ZinchenkoR. A.

Зинченко Рена Абульфазовна – д. м. н., профессор, член-корр. Российской академии наук, заместитель директора по научно клинической работе, заведующая лабораторией генетической эпидемиологии

Author ID: 93625, Researcher ID: А-9554-2016; Author ID (Scopus): 6603422100

115522, Москва, ул. Москворечье, 1, тел.: (499) 324-12-24;

Rena A. Zinchenko, Doctor of Medicine, Professor, Corresponding Member of the Russian Academy of Sciences, Professor, Honored Worker of Science of the Russian Federation, Deputy Director on Scientific and Clinical work; Head of the laboratory of genetic epidemiology

ul. Moskvorechye 1, Moscow, 115522, tel.: (499) 324-12-24 

renazinchenko@mail.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 «Московский областной научно-исследовательский клинический институт имени М.Ф.Владимирского»РоссияFederal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation ; Moscow Regional Research and Clinical Institute (“MONIKI”)Russian FederationФедеральное государственное бюджетное учреждение «Научно-исследовательский институт пульмонологии» Федерального медико-биологического агентства России»РоссияFederal State Budgetary Institution “Pulmonology Scientific Research Institute” under Federal Medical and Biological Agency of Russian FederationRussian 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 Federation202514122025356875882Copyright © Каширская Н.Ю., Амелина Е.Л., Зинченко Р.А., 20252025Каширская Н.Ю., Амелина Е.Л., Зинченко Р.А.Kashirskaya N.Y., Amelina E.L., Zinchenko R.A.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/4824

Муковисцидоз (МВ) – это ограничивающее продолжительность жизни прогрессирующее аутосомно-рецессивное моногенное заболевание. МВ вызван мутациями в гене CFTR, которые приводят к дисфункции хлорных каналов в эпителиальных клетках практически всех экзокринных систем человека, нарушая работу потовых и слюнных желез, экзокринной части поджелудочной железы, гепатобилиарной и репродуктивной систем, кишечника и респираторного тракта. Прорыв в лечении МВ произвела разработка CFTR-модуляторов, обладающих способностью исправлять дисфункцию белка CFTR на клеточном уровне.

Целью обзора являлся поиск ответа на следующие вопросы: действительно ли новый трехкомпонентный препарат ванзакафтор / тезакафтор / дейтивакафтор (ВТД) обеспечивает существенный шаг вперед в лечении пациентов с МВ? Следует ли всем чувствительным пациентам начинать переход с препарата элексакафтор / тезакафтор / ивакафтор (ЭТИ) на ВТД?

Методы

Методы. Проведен анализ всей доступной научной литературы, содержащейся в базах данных PubMed, Scopus и Web of Science, по сравнительной эффективности ВТД и ЭТИ.

Результаты

Результаты. Выявлено, что к настоящему времени (октябрь 2025 г.) нет доказанного клинического преимущества ВТД над ЭТИ. Препараты сопоставимы по улучшению функции легких, количеству обострений бронхолегочного процесса и побочных реакций. По показателям потовой пробы ВТД демонстрирует преимущество в восстановлении функции белка CFTR, указывая на потенциальную возможность раннего восстановления нормального функционирования хлорных каналов и предотвращения развития или прогрессирования МВ, что следует подтвердить в условиях реальной клинической практики. Режим дозирования ВТД 1 раз в день можно было бы рассматривать как предпочтительное свойство препарата, однако его высокая стоимость не дает этических аргументов в пользу массового перехода с ЭТИ на ВТД ради общественной выгоды. ВТД может быть назначен пациентам 6 лет и старше, имеющих по крайней мере 1 из 31 дополнительных патогенных вариантов, не входящих в список ЭТИ по инструкции.

Заключение

Заключение. Целесообразность отмены успешного патогенетического лечения препаратом ЭТИ и назначения ВТД требует дополнительного изучения в условиях реальной клинической практики.

Cystic fibrosis (CF) is a progressive, life-limiting, autosomal recessive monogenic disease caused by mutations in the CFTR gene, leading to dysfunction of chloride channels in epithelial cells of almost all human exocrine systems, disrupting the function of sweat and salivary glands, the exocrine part of the pancreas, the hepatobiliary and reproductive systems, the intestines, and the respiratory tract. The development of CFTR modulators has revolutionized the treatment of CF by correcting CFTR protein dysfunction at the cellular level.

The aim

The aim. In our review, we tried to answer the question of whether the new three-component modulator vanzacaftor/tezacaftor/deutivacaftor (VTD) represents a significant step forward in the treatment of people with CF and whether all susceptible patients should switch from elexacaftor/tezaftor/ivacaftor (ETI) to VTD.

Methods

Methods. We analyzed all available scientific literature on the comparative efficacy of VTD and ETI. The search was conducted in the PubMed, Scopus, and Web of Science databases.

Results

Results. It has been found that to date (October 2025) there is no proven clinical superiority of VTD over ETI. The drugs are comparable in terms of improving lung function, the number of exacerbations of the bronchopulmonary process, and the number of adverse reactions. Based on sweat test results, VTD demonstrates an advantage in restoring CFTR protein function, indicating the potential for early restoration of normal chloride channel function and prevention of the development or progression of cystic fibrosis, which still needs to be confirmed in real-world clinical practice. The once-daily dosing regimen of VTD could be considered a desirable feature of the drug, but its high cost does not provide ethical arguments in favor of a mass switch from ETI to VTD for the public health benefit. VTD can be prescribed to patients aged 6 years and older who have at least one of 31 additional pathogenic variants not listed in the prescribing information for ETI.

Conclusion

Conclusion. The advisability of discontinuing successful pathogenetic treatment with ETI and prescribing VTD requires further study in real clinical practice.

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