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Physiology in Medicine review article. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem-cell disorder caused by a somatic mutation in a gene known as phosphatidylinositol glycan class A (PIGA). It may arise de novo or in the setting of acquired aplastic anemia.The absence of GPI-anchored proteins leads to complement-mediated intravascular hemolysis, because 2 important complement regulatory proteins (CD55 and CD59) are missing from PNH cells. Therapeutic options include supportive care, bone marrow transplantation, and monoclonal antibody therapy with the terminal complement inhibitor eculizumab.
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