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
收藏
页码：1126 / 1129
页数：4
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