Data presented at the 14th annual meeting of the European Hematology Association, in Berlin, Germany, show that US drugmaker Alexion's Soliris (eculizumab), a first-in-class terminal complement inhibitor, reduced hemolysis and improved symptoms in nine patients with paroxysmal nocturnal hemoglobinuria who had received no blood transfusions prior to initiating Soliris therapy.
In a second analysis presented at the EHA conference, investigators assessed the safety and efficacy of Soliris in the treatment of nine patients with PNH who required no transfusions prior to starting therapy.
All patients experienced a dramatic reduction in hemolysis following treatment with Soliris for a median of 16 months, as measured by a median reduction in LDH from 1,500 U/L before treatment to 356 U/L after treatment (p=0.008). Overall, hemoglobin levels increased significantly from 9.0 g/dL before treatment to 10.7 g/dL after therapy (p=0.0003), with a median increase of 2.0g/dL. The investigator noted that patients reported a marked improvement in quality of life. No serious adverse events were reported.
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