Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment.
Keywords: PNH; complement cascade; complement inhibitors.
Current treatment options for paroxysmal nocturnal hematuria and prices Treatment options for paroxysmal nocturnal hematuria have changed significantly since 2007, when the first medication for this disease was approved in the United States. This review begins by explaining the biochemical pathways that explain the pathophysiology of paroxysmal nocturnal hemoglobinuria. Then, various effects of the disease and treatments including breakthrogh intravascular hemolysis and extravascular hemolysis are explained. Finally, each treatment option for paroxysmal nocturnal hemoglobinuria is discussed and compared in terms of effectiveness, price, and how often the patient needs to take the medication.
© The Author(s), 2024.