The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria
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DOI：
10.1056/nejmoa061648
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发表时间：
2006-09-21
影响因子：
158.5
通讯作者：
Luzzatto, Lucio
中科院分区：
文献类型：
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作者：
Hillmen, Peter;Young, Neal S.;Luzzatto, Lucio
背景：我们测试了依库珠单抗（一种抗补体终末蛋白C5的人源化单克隆抗体，可抑制补体终末活化）治疗阵发性睡眠性血红蛋白尿症（PNH）患者的安全性和有效性。患者接受安慰剂或依库珠单抗静脉给药;依库珠单抗以每周600 mg的剂量给药4周，1周后以900 mg剂量给药，然后每隔一周900 mg，直至第26周。两个主要终点是血红蛋白水平的稳定和输注的浓缩红细胞的单位数。结果：87例患者接受随机分组。在没有输血的情况下，49%（21/43）的依库珠单抗组患者的血红蛋白水平达到稳定，而安慰剂组患者中没有（0/44）达到稳定（P
BACKGROUND:We tested the safety and efficacy of eculizumab, a humanized monoclonal antibody against terminal complement protein C5 that inhibits terminal complement activation, in patients with paroxysmal nocturnal hemoglobinuria (PNH).METHODS:We conducted a double-blind, randomized, placebo-controlled, multicenter, phase 3 trial. Patients received either placebo or eculizumab intravenously; eculizumab was given at a dose of 600 mg weekly for 4 weeks, followed 1 week later by a 900-mg dose and then 900 mg every other week through week 26. The two primary end points were the stabilization of hemoglobin levels and the number of units of packed red cells transfused. Biochemical indicators of intravascular hemolysis and the patients' quality of life were also assessed.RESULTS:Eighty-seven patients underwent randomization. Stabilization of hemoglobin levels in the absence of transfusions was achieved in 49% (21 of 43) of the patients assigned to eculizumab and none (0 of 44) of those assigned to placebo (P