Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening hematologic disorder due to clonal expansion of mutated bone marrow stem cells, in which their progeny lose the capacity to bind proteins to the cell surface. Chronic and/or paroxysmal intravascular hemolysis and predilection to thrombosis are caused by the loss of the complement inhibitors CD55 and CD59 on the surface of red blood cells (RBC). A fraction of patients may also develop clinically severe aplastic anemia or myelodysplastic syndrome, as well as hypocellular or dysplastic bone marrow. PNH is characterized by fatigue, dysphagia, abdominal pain, dyspnea, dark urine, and erectile dysfunction. It predominantly affects Asians between 30 and 59 years. Therapy for PNH is evolving rapidly with the availability of biologic therapies that target the underlying complement-mediated hemolysis.

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Published in: Health Science Reports
License: http://creativecommons.org/licenses/by/4.0/
See article on publisher's website: https://dx.doi.org/10.1002/hsr2.512