| abstract |
The present invention provides treatment of patients with paroxysmal nocturnal hemoglobinuria with inhibitors of complement. Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, is active in a preliminary 12-week open label study in a small cohort of patients with paroxysmal nocturnal hemoglobinuria (PNH) It was. This is a multicenter, double-blind, randomized, placebo-controlled, international phase III study, where long-term eculizumab treatment can reduce intravascular hemolysis or stabilize hemoglobin levels Whether the need for blood transfusion can be reduced and the quality of life can be improved. Eculizumab was found to stabilize hemoglobin levels, reduce the need for blood transfusions, and improve the quality of life of PNH patients by reducing intravascular hemolysis. [Selection figure] None |