Loading...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare but devastating hematologic disorder caused by an acquired defect in the binding of complement regulatory proteins to the surface of red blood cells. As a consequence, these red blood cells are susceptible to complement-mediated hemolysis. In addition to having anemia, patients with PNH often experience thrombosis, particularly in the splanchnic bed. Recently, a humanized monoclonal antibody (eculizumab [Soliris]) that targets complement protein C5 has been approved for the treatment of patients with PNH.
To evaluate the effectiveness of eculizumab for preventing thromboembolism (TE) in PNH patients, researchers from several universities and the NIH examined data on 195 participants in thre…