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

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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»

Автор, ответственный за переписку.
ORCID iD: 0000-0003-1878-4467
Россия

Б. А. Бакиров

ФГБОУ ВО «Башкирский государственный медицинский университет» Минздрава России

Email: bakirovb@gmail.com
ORCID iD: 0000-0002-3297-1608

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

450075, Уфа, ул. Блюхера, 3 

Россия

В. Н. Павлов

ФГБОУ ВО «Башкирский государственный медицинский университет» Минздрава России

ORCID iD: 0000-0003-2125-4897
450075, Уфа, ул. Блюхера, 3 Россия

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