Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disease whose main symptom is complement-mediated intravascular hemolysis as a result of the clonal expansion of hematopoietic stem cells having mutations in genes involved in glycosylphosphatidylinositol (GPI) anchor synthesis including PIGA. With the advent of a humanized anti-C5 monoclonal antibody (eculizumab), the inhibitory effect on hemolysis, improvement in its various complicating symptoms, and preventive effect on thrombus formation were observed. In addition, the QOL in patients with PNH was significantly improved. Subsequently, the technology of recycling antibodies (ravulizumab and crovalimab) significantly extended the treatment interval and improved convenience, although the poor improvement of anemia due to extravascular hemolysis has been a major issue in some patients. Several clinical trials using proximal complement inhibitors (C3, factor D, factor B) are being conducted to overcome this critical task. Not only efficacy but also safety and convenience will be evaluated, and the best therapeutic agent will be selected in the near future.
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