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Med Sci (Paris). 2009 Dec;25(12):1126-9. doi: 10.1051/medsci/200925121126.

[Eculizumab in paroxysmal nocturnal hemoglobinuria].

[Article in French]

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  • 1Service d'hématologie-greffe, Inserm U728, Hôpital Saint-Louis, AP-HP, 1, avenue Claude Vellefaux, 75010 Paris, France. gerard.socie@sls.aphp.fr


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.

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