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Vol. 22, Art. 12 (pp. 172-181)    |    2021       

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Changes in the parameters of the hemostatic system in patients with paroxysmal nocturnal hemoglobinuria during eculizumab therapy

Russian Research Institute of Hematology and Transfusiology

Brief summary

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, progressive and disabling systemic disease with life-threatening complications. PNH is characterized by chronic complement-dependent hemolysis, which causes the risk of developing such severe conditions as arterial and venous thrombosis. In all patients with PNH we examined, the prothrombotic state was characterized by an increase in the activity of factor VIII and the level of D-dimer, as well as the functional activity of platelets in patients with the most pronounced signs of hypercoagulability. Screening tests (activated partial thromboplastin time, prothrombin according to Quick, the amount of fibrinogen), as well as the activity of natural anticoagulants - antithrombin, protein C and the level of protein S were within the normal range. To date, eculizumab is the only effective means of pathogenetic therapy for PNH and is a monoclonal antibody that blocks the formation of the terminal complement activation complex at the stage of activation of the C5 component and the formation of a membrane-attacking complex (MAC). Considering that a prothrombotic state develops in PNH, in order to assess the therapeutic efficacy of eculizumab, it was important to assess its effect on the state of the hemostasis system. The study showed that eculizumab treatment already within the first months has a normalizing effect on the hemostasis system of patients with PNH, as evidenced by a decrease in the D-dimer level, factor VIII activity and the functional activity of platelets to normal values.


Key words

paroxysmal nocturnal hemoglobinuria (PNH), complement-dependent hemolysis, thrombotic complications, hemostasis, eculizumab.

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