Eculizumab in paroxysmal nocturnal hemoglobinuria

被引：0

作者：

Socie, Gerard ^{[1
]}

Varoqueaux, Nathalie ^{[2
]}

de Latour, Regis Peffault ^{[1
]}

机构：

[1] Hop St Louis, INSERM, AP HP, Serv Hematol Greffe,U728, F-75010 Paris, France

[2] Alexion Pharma France, Direct Affaires Med, F-75008 Paris, France

来源：

M S-MEDECINE SCIENCES | 2009年 / 25卷 / 12期

关键词：

COMPLEMENT INHIBITOR ECULIZUMAB; PIG-A GENE; MICROPARTICLES; HEMOLYSIS; DISEASE;

D O I：

10.1051/medsci/200925121126

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Eculizumab in paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria is a rare acquired clonal of the hematopoietic stem cell due to acquired mutation of the PIG-A gene. This results in the lack of two GPI-anchored membrane proteins involved in the inhibition of complement attack, thus explaining red cells hemolysis. The development of an anti-C5 monoclonal antibody (eculizumab) had profoundly modified the treatment of the the hemolytic form of the disease.

引用

页码：1126 / 1129

页数：4

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