Pediatric Hematology/Oncology and ImmunopathologyPediatric Hematology/Oncology and ImmunopathologyВопросы гематологии/онкологии и иммунопатологии в педиатрии1726-17082414-9314Fund Doctors, Innovations, Science for Children39410.24287/1726-1708-2020-19-3-131-138LITERATURE REVIEWОБЗОР ЛИТЕРАТУРЫModern approaches in hemophilia therapyСовременные подходы к лечению гемофилииhttps://orcid.org/0000-0003-4555-9337FlorinskiyD. B.ФлоринскийД. Б.
Russian Federation

resident in pediatrics, 

117997, Moscow, Samory Mashela st., 1

врач-ординатор,

117997, Москва, ул. Саморы Машела, 1

mitia94@yandex.ruhttps://orcid.org/0000-0003-4384-6754ZharkovP. A.ЖарковП. А.
Russian Federation

117997, Moscow, Samory Mashela st., 1

117997, Москва, ул. Саморы Машела, 1

Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Ministry of Healthcare of Russian FederationФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Минздрава России080920201931311380810202008102020Copyright ©; 2025, «D. Rogachev NMRCPHOI»Copyright ©; 2025, ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России2025«D. Rogachev NMRCPHOI»ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава Россииhttps://creativecommons.org/licenses/by/4.0https://hemoncim.com/jour/article/view/394

For 60 years the “gold standard” of hemophilia treatment has been the use of concentrates factors. In recent years, there has been a rapid development of therapeutics. Several extended half-life concentrates have been introduced into clinical practice, as well as a non – factorial type of therapy-a bispecific antibody mimicking activated factor VIII. Encouraging results in clinical trials show the use of antibodies to tissue factor pathway inhibitor, small interfering RNA to antithrombin and gene therapy. The advantages of new treatment methods are a reduction in the frequency of intravenous injections and improved adherence to therapy, especially in patients with inhibitors. Long-term safety and effectiveness require further study.

«Золотым стандартом» терапии гемофилии уже 60 лет остается применение концентратов факторов. В последние годы отмечается бурное развитие опций лечения. В клиническую практику внедрены концентраты с длительным периодом полувыведения, а также применяется нефакторный вид терапии – биспецифическое антитело, имитирующее функцию активированного фактора VIII. Обнадеживающие результаты в клинических исследованиях показывает применение антител к ингибитору пути тканевого фактора, интерферирующего агента РНК, ингибирующего антитромбин, а также генной терапии. Преимуществами новых методов лечения является сокращение частоты внутривенных инъекций и улучшение приверженности терапии, особенно у пациентов с ингибиторной формой гемофилии. Долгосрочная безопасность и эффективность требуют дальнейшего изучения.

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