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Hemophilia: the role of national drugs for substitution therapy in pediatric medical practice

DOI: https://doi.org/10.29296/25877305-2022-04-06
Issue: 
4
Year: 
2022

Professor K. Grigoryev, MD; T. Plahuta, Candidate of Medical Sciences;
A. Solovieva, Candidate of Medical Sciences; E. Vartapetova, Candidate
of Medical Sciences;
N.I. Pirogov Russian National Research Medical University, Moscow

Hemophilia is one of the most common genetically determined blood clotting disorders. The disease manifests itself in early childhood and clinically has two of the most common variants of the disease: hemophilia A – deficiency of factor (F) VIII, and hemophilia B – deficiency of FIX. Diagnostics requires obligatory laboratory confirmation of the genetic absence or decrease in the concentration of FVIII for hemophilia A and FIX for hemophilia B. Introduction in recent years into practice of domestic recombinant coagulation factors – moroctocog alfa (BDDrFVIII), nonacog alfa (rFIX) and eptacog alfa (activated) (rFVIIa) made it possible to significantly optimize substitution therapy in children with hemophilia, and in the face of growing Western sanctions, to provide Russian children with the necessary means of emergency care and prevention, which are not inferior to the best foreign analogues. Their high therapeutic efficacy and low immunogenicity have been proven. New therapeutic principles include non-factorial drugs and gene therapy options for both hemophilia A and hemophilia B.

Keywords: 
hemophilia
children
diagnostics
moroctocog alfa
nonacog alfa
eptacog alfa (activated).



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