Renal Transplantation Under Prophylactic Eculizumab in Atypical Hemolytic Uremic Syndrome With CFH/CFHR1 Hybrid Protein
Renal Transplantation Under Prophylactic Eculizumab in Atypical Hemolytic Uremic Syndrome With CFH/CFHR1 Hybrid Protein
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DOI：
10.1111/j.1600-6143.2012.04051.x
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发表时间：
2012-07-01
影响因子：
8.8
通讯作者：
Niaudet, P.
中科院分区：
文献类型：
--
作者：
Krid, S.;Roumenina, L. T.;Niaudet, P.
我们报告了在一名患有非典型溶血性尿毒综合征（阿胡斯）和已知CFH/CFHR 1杂合基因的7岁男孩中首次观察到在预先艾库组单抗治疗下成功进行肾移植，该男孩在3年透析期间依赖血浆治疗。杂合CFH/CFHR 1蛋白具有改变的C3 b/C3 d结合，不能保护细胞免受补体攻击，并且直接涉及阿胡斯发病机制。在第一个16个月的随访期间没有复发的证据。我们的结论是，单用依库珠单抗，而不用血浆治疗（血浆输注和/或血浆置换），足以预防阿胡斯复发并维持长期移植物功能。
We report the first observation of successful kidney transplantation under pre-emptive eculizumab treatment in a 7-year-old boy with atypical hemolytic uremic syndrome (aHUS) and a known hybrid CFH/CFHR1 gene, who was dependent on plasma therapy during the 3-year dialysis period. The hybrid CFH/CFHR1 protein has an altered C3b/C3d binding, is incapable to protect cells from complement attack and is directly implicated in aHUS pathogenesis. There was no evidence of recurrence during the first 16-month follow-up period. We conclude that eculizumab alone, without plasma therapy (plasma infusion and/or plasma exchange), is sufficient to prevent recurrence of aHUS and to maintain long-term graft function.