What is the role of complement inhibition in the treatment of paroxysmal nocturnal hemoglobinuria?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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Three monoclonal antibodies that target complement have been approved for use in PNH. Eculizumab and ravulizumab are humanized monoclonal antibodies that target terminal complement protein C5. Both agents have been shown to decrease intravascular hemolysis, reduce the need for blood transfusions, and improve PNH-related symptoms such as fatigue. [37, 38, 39, 40]  Both are administered intravenously, but eculizumab is infused IV every 2 weeks and ravulizumab is infused every 8 weeks. Pegcetacoplan targets complement protein C3, and can control both intravascular and extravascular hemolysis.

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