C3-mediated extravascular hemolysis in PNH on eculizumab: mechanism and clinical implications

The introduction of eculizumab, a human monoclonal antibody against the C5 component of complement, has changed radically the management of paroxysmal nocturnal hemoglobinuria (PNH). The blockade of the terminal complement pathway by eculizumab eculizumab abrogates intravascular hemolysis, reduces the transfusion requirement and the risk of thrombosis in most of hemolytic PNH patients. However, in almost all PNH patients on eculizumab arises a fraction of PNH red cells that bind fragments of C3 and become a potential target of phagocytosis by macrophages.

Source: Seminars in Hematology - June 5, 2018 Category: Hematology Authors: Rosario Notaro, Michela Sica Source Type: research