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在第三次肾移植治疗伴 CFH 突变的非典型溶血尿毒症综合征后,使用依库珠单抗治疗并停止血浆置换以维持肾功能。

Maintenance of kidney function following treatment with eculizumab and discontinuation of plasma exchange after a third kidney transplant for atypical hemolytic uremic syndrome associated with a CFH mutation.

机构信息

Paediatric Nephrology Department, Emma Children's Hospital/Academic Medical Centre of Amsterdam, Amsterdam Zuid-Oost, The Netherlands.

出版信息

Am J Kidney Dis. 2010 Apr;55(4):708-11. doi: 10.1053/j.ajkd.2009.08.011. Epub 2009 Oct 25.

Abstract

Kidney transplant in patients with atypical hemolytic uremic syndrome (aHUS) is associated with a poor outcome because of recurrent disease, especially in patients known to have a factor H mutation. Long-term prophylactic plasma exchange and combined liver-kidney transplant have prevented graft loss caused by recurrence. However, the mortality associated with liver transplant is not negligible, and prophylactic plasma exchange requires permanent vascular access and regular hospitalization and exposes the patient to potential allergic reactions to plasma. Eculizumab is a high-affinity humanized monoclonal antibody that binds to C5 and thus prevents generation of C5a and the membrane attack complex. We report the case of a 17-year-old girl with aHUS associated with a mutation in the gene for complement factor H (CFH; c.3572C>T, Ser1191Leu) who was highly dependent on plasma exchange. Because of severe allergic reactions to plasma after the third renal graft, eculizumab was introduced in place of plasma exchange without problems. This and other reports suggest that the promise of complement inhibitors in the management of aHUS is going to be fulfilled.

摘要

在补体因子 H 基因突变的非典型溶血性尿毒症综合征(aHUS)患者中,肾移植后疾病易复发,导致预后不良,特别是在已知存在补体因子 H 突变的患者中。长期预防性血浆置换和肝-肾联合移植可预防因复发导致的移植物丢失。然而,肝移植相关的死亡率不可忽视,且预防性血浆置换需要永久性血管通路和定期住院治疗,使患者面临潜在的血浆过敏反应风险。依库珠单抗是一种高亲和力的人源化单克隆抗体,可与 C5 结合,从而阻止 C5a 和膜攻击复合物的生成。我们报告了一例 17 岁的 aHUS 女孩病例,该患者与补体因子 H 基因(CFH;c.3572C>T,Ser1191Leu)突变有关,对血浆置换高度依赖。由于第三次肾移植后对血浆发生严重过敏反应,依库珠单抗被引入以替代血浆置换,未出现问题。这一病例及其他报告表明,补体抑制剂在 aHUS 治疗中的前景值得期待。

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