Polymorphism of the complement receptor 1 gene correlates with hematological response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria
نویسندگان
چکیده
Citation: Rondelli T, Risitano AM, Peffault de Latour R, Sica M, Peruzzi B, Ricci P, Barcellini W, Iori AP, Boschetti C, Valle V, Frémeaux-Bacchi V, De Angioletti M, Socié G, Luzzatto L, and Notaro R. Polymorphism of the complement receptor 1 gene correlates with hematological response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria. Haematologica. 2013; 98:xxx doi:10.3324/haematol.2013.090001
منابع مشابه
Polymorphism of the complement receptor 1 gene correlates with the hematologic response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria.
Complement blockade by eculizumab is clinically effective in hemolytic paroxysmal nocturnal hemoglobinuria. However, the response is variable and some patients remain dependent on red blood cell transfusions. In 72 patients with hemolytic paroxysmal nocturnal hemoglobinuria on eculizumab we tested the hypothesis that response may depend on genetic polymorphisms of complement-related genes. We f...
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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal blood disorder that manifests with hemolytic anemia, bone marrow failure, and thrombosis. Many of the clinical manifestations of the disease result from complement-mediated intravascular hemolysis. Allogeneic bone marrow transplantation is the only curative therapy for PNH. Eculizumab, a monoclonal antibody that blocks terminal compleme...
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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias. The absence of two glycosylphosphatidylinositol (GPI)-anchored proteins, CD55 and CD59, leads to uncontrolled complement activation that accounts for hemolysis and other PNH manifestations. GPI anchor protein deficiency is almost alw...
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Paroxysmal nocturnal hemoglobinuria (PNH) is a hematologic disorder characterized by clonal expansion of red blood cells (RBCs) lacking the ability to inhibit complement-mediated hemolysis. Eculizumab, a humanized monoclonal antibody that binds the C5 complement protein, blocks serum hemolytic activity. This study evaluated the long-term safety and efficacy of eculizumab in 11 patients with PNH...
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