Eirik Tjonnfjord
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare aquired hematopoetic stem cell disorder characterized by the triad of intravascular hemolysis, thrombosis and bone marrow failure. The symptoms are caused by complement activation on the cell surface of hematopoetic cells. The detection of a PNH clone is made by analyzing GPI-anchored molecules on blood cells by flowcytometry. The only curative treatment, allogeneic stem cell transplantation is limited by short- and long-term consequences, and donor availability. Eculizumab, a humanized monocolonal antibody against terminal complement protein C5 is currently the most effective treatment.
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