Novel insights into the treatment of complement-mediated hemolytic anemias

被引:66
作者
Berentsen, Sigbjorn [1 ]
Hill, Anita [2 ]
Hill, Quentin A. [2 ]
Tvedt, Tor Henrik Anderson [3 ]
Michel, Marc [4 ]
机构
[1] Haugesund Hosp, Dept Res & Innovat, POB 2170, N-5504 Haugesund, Norway
[2] Leeds Teaching Hosp, Dept Haematol, Leeds, W Yorkshire, England
[3] Haukeland Hosp, Dept Med, Sect Hematol, Bergen, Norway
[4] Univ Paris Est, Henri Mondor Hosp, AP HP, Dept Med, Creteil, France
关键词
autoimmune hemolytic anemia; cold agglutinin disease; complement; complement inhibitors; paroxysmal nocturnal hemoglobinuria; therapy; COLD AGGLUTININ DISEASE; PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA; INHIBITOR ECULIZUMAB; RED-CELLS; PIG-A; INTRAVASCULAR HEMOLYSIS; NITRIC-OXIDE; AUTOIMMUNE; RITUXIMAB; ERYTHROCYTES;
D O I
10.1177/2040620719873321
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Complement-mediated hemolytic anemias can either be caused by deficiencies in regulatory complement components or by autoimmune pathogenesis that triggers inappropriate complement activation. In paroxysmal nocturnal hemoglobinuria (PNH) hemolysis is entirely complement-driven. Hemolysis is also thought to be complement-dependent in cold agglutinin disease (CAD) and in paroxysmal cold hemoglobinuria (PCH), whereas warm antibody autoimmune hemolytic anemia (wAIHA) is a partially complement-mediated disorder, depending on the subtype of wAIHA and the extent of complement activation. The pathophysiology, clinical presentation, and current therapies for these diseases are reviewed in this article. Novel, complement-directed therapies are being rapidly developed. Therapeutic terminal complement inhibition using eculizumab has revolutionized the therapy and prognosis in PNH but has proved less efficacious in CAD. Upstream complement modulation is currently being investigated and appears to be a highly promising therapy, and two such agents have entered phase II and III trials. Of these, the anti-C1s monoclonal antibody sutimlimab has shown favorable activity in CAD, while the anti-C3 cyclic peptide pegcetacoplan appears to be promising in PNH as well as CAD, and may also have a therapeutic potential in wAIHA.
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页数:20
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