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Современные и разрабатываемые биотехнологические препараты для лечения патологии системы комплемента при пароксизмальной ночной гемоглобинурии

https://doi.org/10.24287/1726-1708-2020-19-3-164-172

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Аннотация

Пароксизмальная ночная гемоглобинурия (ПНГ) – это редкое приобретенное клональное заболевание крови, развивающееся в результате соматической мутации гена фосфатидилинозитол гликана класса А (PIG-A) в стволовых кроветворных клетках и проявляющееся гемолитической анемией, костномозговой недостаточностью, тромбозами, нарушением функции почек и другими тяжелыми клиническими синдромами. Лечение ПНГ является сложной клинической задачей, требующей комплексного подхода. В последнее десятилетие таргетная терапия с применением ингибитора терминального этапа активации комплемента антитела экулизумаб играет ключевую роль в лечении ПНГ. Экулизумаб является первым гуманизированным моноклональным анти-С5-антителом, подтвердившим эффективное ингибирование системы комплемента и одобренным во многих странах в качестве стандарта лечения ПНГ. С 2019 г. в Российской Федерации широко используется для лечения ПНГ первый биоаналог экулизумаба (Элизария), подтвердивший в ходе клинических исследований аналогичные оригинальному препарату эффективность и безопасность. В настоящее время продолжаются разработки новых препаратов ингибиторов комплемента, которые подразделяются по механизму действия на ингибиторы C5-компонента комплемента (терминальная часть) и ингибиторы ранних фаз каскада комплемента (проксимальная часть). К новым препаратам относятся моноклональные антитела, малые молекулы, небольшие пептидные ингибиторы и малые интерферирующие РНК, а также рекомбинантные белки, полученные на основе эндогенных регуляторов активации комплемента.

Об авторах

Д. А. Кудлай
ФГАОУ ВО «Первый Московский государственный медицинский университет им. И.М. Сеченова» Минздрава России (Сеченовский Университет); АО «ГЕНЕРИУМ»
Россия


Б. А. Бакиров
ФГБОУ ВО «Башкирский государственный медицинский университет» Минздрава России
Россия

д-р. мед. наук, доцент, заведующий кафедрой госпитальной терапии №2;

450075, Уфа, ул. Блюхера, 3 



В. Н. Павлов
ФГБОУ ВО «Башкирский государственный медицинский университет» Минздрава России
Россия
450075, Уфа, ул. Блюхера, 3


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Для цитирования:


Кудлай Д.А., Бакиров Б.А., Павлов В.Н. Современные и разрабатываемые биотехнологические препараты для лечения патологии системы комплемента при пароксизмальной ночной гемоглобинурии. Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2020;19(3):164-172. https://doi.org/10.24287/1726-1708-2020-19-3-164-172

For citation:


Kudlay D.A., Bakirov B.A., Pavlov V.N. Biotechnological products for the treatment of complement system disorders including paroxysmal nocturnal hemoglobinuria: currently available and in development. Pediatric Hematology/Oncology and Immunopathology. 2020;19(3):164-172. (In Russ.) https://doi.org/10.24287/1726-1708-2020-19-3-164-172

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ISSN 1726-1708 (Print)
ISSN 2414-9314 (Online)