What are the treatment options for paroxysmal nocturnal hemoglobinuria (PNH)?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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Answer

Pegcetacoplan, eculizumab, and ravulizumab are approved for treatment of PNH. These monoclonal antibodies are targeted inhibitors of complement that alleviate the hemolysis associated with PNH and its sequelae, dramatically improving symptoms, improving quality of life, and eliminating complications of PNH; however, they do not alter the underlying defect of the disease. Allogeneic hematopoietic stem cell transplantation is curative but it requires a histocompatible donor and is associated with significant morbidity and mortality, so it is reserved for severe cases of PNH with aplastic anemia or transformation to leukemia. See Treatment.

For patient education information, see Anemia.

 


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