Department of Neurology, University of North Carolina, 2200 Physicians Office Building, CB 7025, 170 Manning Drive, Chapel Hill, North Carolina, 27599-7025, USA.
Muscle Nerve. 2013 Jul;48(1):76-84. doi: 10.1002/mus.23839. Epub 2013 Apr 30.
Complement activation at the neuromuscular junction is a primary cause of acetylcholine receptor loss and failure of neuromuscular transmission in myasthenia gravis (MG). Eculizumab, a humanized monoclonal antibody, blocks the formation of terminal complement complex by specifically preventing the enzymatic cleavage of complement 5 (C5).
This study was a randomized, double-blind, placebo-controlled, crossover trial involving 14 patients with severe, refractory generalized MG (gMG).
Six of 7 patients treated with eculizumab for 16 weeks (86%) achieved the primary endpoint of a 3-point reduction in the quantitative myasthenia gravis (QMG) score. Examining both treatment periods, the overall change in mean QMG total score was significantly different between eculizumab and placebo (P = 0.0144). After assessing data obtained from all visits, the overall change in mean QMG total score from baseline was found to be significantly different between eculizumab and placebo (P < 0.0001). Eculizumab was well tolerated.
The data suggest that eculizumab may have a role in treating severe, refractory MG.
补体在神经肌肉接头的激活是重症肌无力（MG）中乙酰胆碱受体丢失和神经肌肉传递失败的主要原因。依库珠单抗是一种人源化单克隆抗体，通过特异性阻止补体 5（C5）的酶切来阻止末端补体复合物的形成。
这是一项涉及 14 例严重、难治性全身性 MG（gMG）患者的随机、双盲、安慰剂对照、交叉试验。
16 周接受依库珠单抗治疗的 7 例患者中有 6 例（86%）达到了 QMG 评分降低 3 分的主要终点。在检查两个治疗期时，依库珠单抗和安慰剂之间的平均 QMG 总评分的总体变化有显著差异（P = 0.0144）。在评估所有就诊时的数据后，发现依库珠单抗和安慰剂之间从基线开始的平均 QMG 总评分的总体变化有显著差异（P < 0.0001）。依库珠单抗耐受性良好。
数据表明，依库珠单抗可能在治疗严重、难治性 MG 方面具有作用。
