Hemophilia

Hemophilia is a severe hereditary disorder characterized by a clotting disorder (coagulation) resulting from the absence of clotting factors VIII (hemophilia A) or IX (hemophilia B).

The main manifestations of hemophilia are hemorrhages in muscles, joints and internal organs. Frequent joint hemorrhages lead to early and persistent disability, and severe bleeding or hemorrhage into vital organs can lead to premature death.

Treatment of patients with hemophilia is usually based on substitution therapy, i.e. on the replacement of the missing clotting factor. However, in the body of about a quarter of patients with hemophilia, antibodies form - the so-called. inhibitors that block the activity of missing coagulation factors. In other words, any replacement drug administered will be neutralized by inhibitors and therefore ineffective in trying to stop bleeding.

Studies and awareness of the role of tissue factor (TF) and its activity in the coagulation process after binding to factor VIIa led to the formation of the hypothesis that the TF / FVIIa complex can contribute to normal hemostasis even in the absence of some coagulation factors.

The development and clinical confirmation of the hypothesis was in the 80s of the last century. Research led to the development of recombinant activated factor VII (rFVIIa). This fundamentally new method of treatment has shown high efficiency in hundreds of patients with an inhibitory form of hemophilia and is recognized as the optimal treatment for such patients. The efficacy of rFVIIa does not depend on the inhibitor titer.