biopreparatБИОпрепараты. Профилактика, диагностика, лечениеBiological Products. Prevention, Diagnosis, Treatment2221-996X2619-1156Scientific Centre for Expert Evaluation of Medicinal Products10.30895/2221-996X-2021-21-1-39-49biopreparat-313Research ArticleОБЗОРЫREVIEWSЛекарственные препараты фактора VIII, актуальные вопросы разработки, клинического исследования и применения (часть 1)Factor VIII products: key aspects of development, clinical research and use (part 1)https://orcid.org/0000-0002-9377-1378АвдееваЖ. И.AvdeevaZh. I.

Авдеева Жанна Ильдаровна, доктор медицинских наук, профессор

Петровский б-р, д. 8, стр. 2, Москва, 127051

Zhanna I. Avdeeva, Dr. Sci. (Med.), Professor

8/2 Petrovsky Blvd, Moscow 127051

avdeeva@expmed.ruhttps://orcid.org/0000-0001-6624-2692СолдатовА. А.SoldatovA. A.

Солдатов Александр Алексеевич, доктор медицинских наук

Петровский б-р, д. 8, стр. 2, Москва, 127051

Aleksandr A. Soldatov, Dr. Sci. (Med.)

8/2 Petrovsky Blvd, Moscow 127051

Soldatov@expmed.ruhttps://orcid.org/0000-0001-6472-6386БондаревВ. П.BondarevV. P.

Бондарев Владимир Петрович, доктор медицинских наук, профессор

Петровский б-р, д. 8, стр. 2, Москва, 127051

Vladimir P. Bondarev, Dr. Sci. (Med.), Professor

8/2 Petrovsky Blvd, Moscow 127051

Bondarev@expmed.ruhttps://orcid.org/0000-0003-0269-8337МосягинВ. Д.MosyaginV. D.

Мосягин Вячеслав Дмитриевич, доктор медицинских наук, профессор

Петровский б-р, д. 8, стр. 2, Москва, 127051

Vyacheslav D. Mosyagin, Dr. Sci. (Med.), Professor

8/2 Petrovsky Blvd, Moscow 127051

mosiagin@expmed.ruhttps://orcid.org/0000-0003-4891-973XМеркуловВ. А.MerkulovV. A.

Меркулов Вадим Анатольевич, доктор медицинских наук, профессор

Петровский б-р, д. 8, стр. 2, Москва, 127051

Трубецкая ул., д. 8, стр. 2, Москва, 119991

Vadim A. Merkulov, Dr. Sci. (Med.), Professor

8/2 Petrovsky Blvd, Moscow 127051

8/2 Trubetskaya St., Moscow 119991

merkulov@expmed.ruФедеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской ФедерацииРоссияScientific Centre for Expert Evaluation of Medicinal ProductsRussian FederationФедеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации; Федеральное государственное автономное образовательное учреждение высшего образования «Первый Московский государственный медицинский университет им. И. М. Сеченова» Министерства здравоохранения Российской ФедерацииРоссияScientific Centre for Expert Evaluation of Medicinal Products; I. M. Sechenov First Moscow State Medical UniversityRussian Federation2021200220212113949Copyright © Авдеева Ж.И., Солдатов А.А., Бондарев В.П., Мосягин В.Д., Меркулов В.А., 20212021Авдеева Ж.И., Солдатов А.А., Бондарев В.П., Мосягин В.Д., Меркулов В.А.Avdeeva Z.I., Soldatov A.A., Bondarev V.P., Mosyagin V.D., Merkulov V.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.biopreparations.ru/jour/article/view/313

По информации Всемирной федерации гемофилии, в настоящее время в мире насчитывается около 400 тыс. пациентов с заболеванием гемофилией. Тяжелые клинические проявления заболевания, связанные с генетически детерминированным дефицитом активности фактора свертывания крови, требуют проведения постоянной заместительной терапии препаратами свертывания крови. Длительное применение препаратов белковой природы часто приводит к формированию специфических антител, что вызывает снижение или потерю эффективности лекарственного препарата или является причиной выраженных побочных реакций, вплоть до анафилаксии. В связи с этим актуальным является поиск новых оптимальных подходов к способам лечения гемофилии, что требует разработки новых лекарственных препаратов факторов свертывания крови и совершенствования технологии производства ранее зарегистрированных препаратов, а так и использования препаратов «нефакторной» терапии. Цель работы – представление результатов анализа актуальных вопросов, касающихся разработки и особенностей плазменных и рекомбинантных лекарственных препаратов фактора VIII, новых подходов к лечению гемофилии А, в том числе и использования препаратов «нефакторной» терапии. В обзоре представлены обобщенные современные данные об этиологии, клинических проявлениях, осложнениях, связанных с проводимой терапией гемофилии А. Приведены сведения о лекарственных препаратах факторов свертывания крови (плазменных и рекомбинантных), используемых в качестве заместительной терапии при указанной патологии. Отражены сведения о перспективных разработках новых лекарственных препаратов, базирующихся на современных достижениях биотехнологии. Имеющиеся разработки открывают перспективы успешного применения таких препаратов в клинической практике.

According to the World Federation of Hemophilia (WFH), there are currently about 400 thousand patients with hemophilia in the world. Severe clinical manifestations of the disease associated with a genetically determined deficiency of blood clotting factor activity require continuous replacement therapy with blood clotting medicines. Long-term use of protein-based medicines often leads to the formation of specific antibodies, which causes a decrease in or loss of efficacy of the medicine or results in severe adverse reactions, including anaphylaxis. Therefore, it is important to search for new optimal approaches to hemophilia treatment, which requires the development of new blood clotting factor products, improvement of the production technology for already authorised products, as well as the use of non-factor products. The aim of the study was to present the results of the analysis of key issues related to the development and characteristics of plasma-derived and recombinant factor VIII products, new approaches to hemophilia A treatment, including the use of non-factor products. The review summarises current data on the etiology, clinical manifestations, and complications of hemophilia A treatment. It provides information on the blood clotting factor products (plasma-derived and recombinant) used as replacement therapy. It also provides information on advanced research projects for the development of new biotechnology-derived products which have good prospects of successful clinical use.

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