Hemophilia in children - modern paradigms for treatment and prevention

Abstract

April 17 marks World Hemophilia Day. The date is dedicated to the birthday of Frank Schnabel, the founder of the World Federation of Hemophilia. And 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 ones in hematology. Hemophilia, although one of the most common genetically determined blood coagulation disorders, is considered a RARE (orphan) disease. The disease manifests itself in early childhood, clinically has two of the most common variants of the disease: hemophilia A and hemophilia B. Diagnosis requires mandatory laboratory confirmation of the genetic absence or decrease in the concentration of FVIII for hemophilia A and FIX for hemophilia B. In recent years, the introduction of domestic recombinant coagulation factors into practice has made it possible to significantly optimize replacement therapy in children with hemophilia, and in the context of increasing Western sanctions, to ensure children of Russia with the necessary means of urgent care and prevention, which are not inferior to the best foreign analogues. New therapy principles that are actually changing the principles of treatment include non-factor drugs and gene therapy options for both hemophilia A and hemophilia B.