| abstract |
PROBLEM TO BE SOLVED: To provide a treatment for a patient with paroxysmal nocturnal hemochromatosis with a complement inhibitor. Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, is a preliminary 1 in a small cohort of patients with paroxysmal nocturnal hemochromatosis (PNH). It showed activity in a 2-week open label test. This trial was a multicenter, double-blind, randomized, placebo-controlled, international, phase III study that received long-term treatment with eculizumab. We investigated whether intravascular hemolysis could be reduced, hemoglobin levels could be stabilized, the need for blood transfusions could be reduced, and quality of life could 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 diagram] None |