Hemophilia in Children - Modern Paradigms of Treatment and Prevention
Keywords:
children, hemophilia, inhibitor, substitution therapy, non-factorial drugsAbstract
World Hemophilia Day is celebrated on April 17. The date is timed to coincide with the birthday of Frank Schneibel, founder of the World Hemophilia Federation. Both on this day and on other days, the medical community is called upon to draw attention to the problems of people suffering from blood clotting disorders — one of the central problems in hematology. Although hemophilia is one of the most common genetically determined disorders of blood clotting, it is considered a rare (orphan) disease. The disease manifests itself in early childhood, has clinically the two most common variants of the disease: hemophilia A and hemophilia B. Diagnosis requires mandatory genetic confirmation or laboratory confirmation of a decrease in the concentration of factor VIII for hemophilia A and factor IX for hemophilia B. The introduction of domestic recombinant coagulation factors in recent years has made it possible to significantly optimize replacement therapy in children with hemophilia, and in the face of growing Western sanctions, to provide Russian children with the necessary urgent care and prevention tools that are not inferior to the best foreign analogues. New principles of therapy that radically change management include non-active drugs and gene therapy options for both hemophilia Aand hemophilia B.
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