The aim of this article is to inform about current situation in the treatment of hemophilia in children. It focuses on substitution therapy, home-based and prophylactic.
In the chapter about substitution therapy we describe basic principles of hemophilia and blood-coagulation factors VIII, IX prepared from plasma or by recombinant technology, we analyse tolerability of these factors in the point of view of microbiological safety and unwanted immunogenicity. Pharmacokinetics of blood-coagulation factors VIII and IX is described as well.
In the chapter about home-based therapy and preventive therapy we describe advances of these therapies. Finally, we describe use of recombinant and plasma-derived factors in Europe and compare it with the situation in the Czech Republic.