人源型抗C5单克隆抗体Eculizumab的应用

Applications of Eculizumab,a humanized anti-complement factor C5 monoclonal antibody

补体系统是获得性免疫系统的重要部分,补体在多种疾病的病理生理过程中发挥着主要作用。阵发性睡眠性血红蛋白尿症(PNH)是一种获得性造血干细胞基因突变所致的难治性溶血性疾病。人源型抗补体蛋白C5单克隆抗体(Eculizumab)是于2007年被美国FDA和欧盟委员会唯一认可的定向治疗PNH的药物。本文将对其在PNH及其他补体相关疾病的治疗中的应用进行综述。

The complement system is an impor- tant part of the innate immune system. Complement plays a crucial role in the pathophysiology of many disorders. Paroxysmal nocturnal hemoglobinuria （PNH） is a refractory hemolytic disease caused by acquired hematopoietic stem cell gene mutation. Ec- ulizumab is a humanized monoclonal antibody that inhibits complement factor C5. It was approved as the only recognition of directional drug for the treat-ment of PNH by the US FDA and the European Commission in 2007. In this article, the applica- tions of Eculizumab in treating PNH and other com- plement related diseases in recent years will be re- viewed.