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Eculizumab (Soliris), a monoclonal antibody directed against complement protein 5, is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Previous trials have shown that the drug decreases hemolysis and reduces the need for transfusions (JW Oncol Hematol Jan 8 2008), but only now is there evidence to show that it also improves survival.
In this report, investigators describe 79 PNH patients who were treated with eculizumab at a single center in the U.K. between 2002 and 2010. Prior to treatment, most patients had transfusion-dependent hemolysis or thrombosis, and 11 had aplastic anemia. Eculizumab was dosed at 600 mg weekly for the first 4 weeks and then at 900 mg every 2 weeks thereafter; five patients required doses o…