Abstract
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.
Highlights
According to the World Federation of Hemophilia (WFH), there are currently about 400 thousand patients with hemophilia in the world
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
Summary
В основе механизмов, определяющих развитие гемофилии, лежат нарушения генов, кодирующих факторы свертывания крови, что является причиной снижения продукции факторов коагуляции крови (VIII, IX или XI) или полное отсутствие их синтеза. Кодирующие продукцию факторов VIII и IX (Xq28 и Xq27 соответственно), локализуются в Х‐хромосоме, их мутация и обусловливает развитие указанной наследственной патологии. Также причиной развития гемофилии может быть не наследственная, а спонтанная мутация генов, кодирующих продукцию VIII или IX факторов коагуляции крови 3. Выделяют три типа гемофилии: гемофилия типа А встречается наиболее часто (в 85% всех случаев), обусловлена дефицитом фактора VIII (FVIII); гемофилия типа В встречается в 13% случаев, обусловлена недостаточной активностью фактора IX (FIX); гемофилия типа С наблюдается в 1–2% случаев, развивается из-за недостаточности фактора XI (FXI). У пациентов часто развиваются подкожные или внутримышечные гематомы, кровоизлияния в суставы даже при незначительных травмах, реже кровоизлияния в брюшную полость, желудочно-кишечные кровотечения, формирование забрюшинных гематом, гематурия и др. Частота встречаемости кровотечений при гемофилии 4 Table 1.
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