Institute of Oncology, St. James's University Hospital, Leeds, UK.
Ther Clin Risk Manag. 2009;5:911-21. doi: 10.2147/tcrm.s3334.
Paroxysmal nocturnal hemoglobinuria is a rare disorder of hemopoietic stem cells. Affected individuals have a triad of clinical associations - intravascular hemolysis, an increased risk of thromboembolism, and bone marrow failure. Most of the symptoms experienced in this disease occur due to the absence of complement regulatory proteins on the surface of the red blood cells. Complement activation is thus not checked and causes destruction of these cells. Eculizumab is a monoclonal antibody treatment which specifically binds to the complement protein C5, preventing its cleavage, and so halts the complement cascade and prevents the formation of the terminal complement proteins. Eculizumab prevents intravascular hemolysis, stabilizes hemoglobin levels, reduces or stops the need for blood transfusions, and improves fatigue and patient quality of life as well as reducing pulmonary hypertension, decreasing the risk of thrombosis and protecting against worsening renal function. It is not a curative therapy but has a great benefit on those with this rare debilitating condition.
阵发性睡眠性血红蛋白尿是一种造血干细胞罕见疾病。受影响的个体有三联临床关联——血管内溶血、血栓栓塞风险增加和骨髓衰竭。这种疾病的大多数症状是由于红细胞表面缺乏补体调节蛋白引起的。因此，补体激活不受控制，导致这些细胞的破坏。依库珠单抗是一种单克隆抗体治疗药物，它特异性地结合补体蛋白 C5，阻止其裂解，从而阻断补体级联反应，防止末端补体蛋白的形成。依库珠单抗可预防血管内溶血，稳定血红蛋白水平，减少或停止输血需求，并改善疲劳和患者生活质量，同时降低肺动脉高压、减少血栓形成风险并防止肾功能恶化。它不是一种治愈性治疗方法，但对患有这种罕见且使人虚弱的疾病的患者有很大益处。
