vspВопросы современной педиатрииCurrent Pediatrics1682-55271682-5535Издательство «ПедиатрЪ»10.15690/vsp.v20i6S.2363vsp-2794Research ArticleОБЗОРЫ ЛИТЕРАТУРЫLITERATURE REVIEWКлиническая эффективность и безопасность комбинированного препарата ивакафтор/лумакафтор у пациентов с муковисцидозом: обзор международных исследованийClinical Efficacy and Safety of Ivacaftor/Lumacaftor Combination in Patients with Cystic Fibrosis: International Studies Reviewhttps://orcid.org/0000-0003-0503-6371КаширскаяН. Ю.KashirskayaNataliya Yu.

Каширская Наталия Юрьевна, доктор медицинских наук, профессор, главный научный сотрудник лаборатории генетической эпидемиологии ФГБНУ «Медико-генетический научный центр имени академика Н.П. Бочкова»; профессор кафедры педиатрии факультета усовершенствования врачей МОНИКИ им. М.Ф. Владимирского

115522, Москва, ул. Москворечье, д. 1

Moscow

kashirskayanj@mail.ruhttps://orcid.org/0000-0001-5933-6594ПетроваН. В.PetrovaNika V.

Москва

Moscow

https://orcid.org/0000-0003-3586-3458ЗинченкоР. А.ZinchenkoRena A.

Москва

Moscow

Медико-генетический научный центр имени академика Н.П. Бочкова; Московский областной научно-исследовательский клинический институт им. М.Ф. ВладимирскогоРоссияResearch Centre for Medical Genetics; Moscow Regional Research and Clinical InstituteRussian FederationМедико-генетический научный центр имени академика Н.П. БочковаРоссияResearch Centre for Medical GeneticsRussian FederationМедико-генетический научный центр имени академика Н.П. Бочкова; Национальный научно-исследовательский институт общественного здоровья имени Н.А. Семашко; РНИМУ им. Н.И. ПироговаРоссияResearch Centre for Medical Genetics; N.A. Semashko National Research Institute of Public Health; Pirogov Russian National Research Medical UniversityRussian Federation202122122021206s558566Copyright © Каширская Н.Ю., Петрова Н.В., Зинченко Р.А., 20212021Каширская Н.Ю., Петрова Н.В., Зинченко Р.А.Kashirskaya N.Y., Petrova N.V., 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://vsp.spr-journal.ru/jour/article/view/2794

Муковисцидоз — аутосомно-рецессивное заболевание, вызванное нарушением структуры гена трансмембранного регулятора проводимости муковисцидоза (CFTR), характеризующееся тяжелым течением и неблагоприятным прогнозом при отсутствии или неоптимальном лечении. Одобрение для клинического применения в 2012 г. в США препаратов патогенетической терапии — модуляторов белка CFTR (потенциатор и корректоры) снизило летальность, связанную с этим заболеванием. В статье представлен обзор исследований клинической эффективности и безопасности комбинированного препарата ивакафтор плюс лумакафтор (Ива/Лум) — первого лицензированного препарата — модулятора CFTR для пациентов-гомозигот по варианту F508del. Показано, что Ива/Лум повышает функцию легких, снижает количество обострений бронхолегочного процесса, в том числе требующих введения антибиотиков и госпитализации, частично восстанавливает экзокринную функцию поджелудочной железы, увеличивает массу тела и массо-ростовой индекс, повышает качество жизни, что позволяет говорить о его благоприятном влиянии на течение и прогноз муковисцидоза. Отмечено также, что раннее начало применения препарата (с двухлетнего возраста) положительно влияет на прогноз заболевания, увеличивая продолжительность жизни и улучшая ее качество.

Cystic fibrosis is an autosomal recessive disease caused by structure abnormalities in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is characterized by severe course and poor prognosis without or with insufficient treatment. Approval of pathogenetic therapy medications, CFTR modulators (potentiators and correctors), for clinical use in 2012 in the United States has reduced mortality from this disease. This article provides the overview of studies on clinical efficacy and safety of ivacaftor/lumacaftor combination (Iva/Lum) — the first licensed CFTR modulator medication for homozygous patients with F508del variant. It was shown that Iva/Lum increases lung function, reduce the number of acute conditions of bronchopulmonary process (including those that require antibiotics and hospitalization), partially restores pancreas exocrine function, increases body weight and mass growth index, and improves quality of life. It allows considering it as favorable effect on the course and prognosis of cystic fibrosis. It was also noted that the early onset of the drug administration (from the age of two) positively affects the prognosis of the disease, increasing life expectancy and improving quality of life.

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