Лекарственные препараты фактора VIII, актуальные вопросы разработки, клинического исследования и применения (часть 1)

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.

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