| 摘要 |
PROBLEM TO BE SOLVED: To provide treatment of paroxysmal nocturnal hemoglobinuria patients by an inhibitor of a complement.SOLUTION: Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, showed activity in a preliminary 12-week open-label trial in a small cohort of patients with paroxysmal nocturnal hemoglobinuria (PNH). The present study has examined whether chronic eculizumab therapy can reduce intravascular hemolysis, stabilize hemoglobin levels, reduce transfusion requirements, and improve quality of life in a double-blind, randomized, placebo-controlled, multi-center global Phase III trial. It is found that eculizumab has stabilized hemoglobin levels, decreased the need for transfusions, and improved quality of life in PNH patients via reduced intravascular hemolysis.SELECTED DRAWING: None |
