• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

依库珠单抗治疗补体因子 B 获得性功能突变所致非典型溶血尿毒综合征

Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B.

机构信息

Regional Paediatric Nephro-Urology Unit, Southampton Children's Hospital, Tremona Road, Southampton, SO16 6YD, UK.

出版信息

Pediatr Nephrol. 2013 Aug;28(8):1315-8. doi: 10.1007/s00467-013-2492-x. Epub 2013 Apr 28.

DOI:10.1007/s00467-013-2492-x
PMID:23624872
Abstract

BACKGROUND

Atypical haemolytic uraemic syndrome (aHUS) is caused by dysregulated complement activation. A humanised anti-C5 monoclonal antibody has recently become available for treatment of this condition

CASE-DIAGNOSIS/TREATMENT: We present the first description of an infant with an activating mutation of complement factor B successfully treated with eculizumab. On standard doses she had evidence of ongoing C5 cleavage despite a good clinical response.

CONCLUSIONS

Eculizumab is effective therapy for aHUS associated with factor B mutations, but recommended doses may not be adequate for all patients.

摘要

背景

非典型溶血尿毒综合征（aHUS）是由补体激活失调引起的。一种人源化抗 C5 单克隆抗体最近已可用于治疗这种疾病。

病例诊断/治疗：我们首次描述了一例成功接受依库珠单抗治疗的补体因子 B 激活突变的婴儿。尽管临床反应良好，但她在标准剂量下仍有持续 C5 裂解的证据。

结论

依库珠单抗是治疗与因子 B 突变相关的 aHUS 的有效疗法，但推荐剂量可能不适用于所有患者。

相似文献

1
Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B.依库珠单抗治疗补体因子 B 获得性功能突变所致非典型溶血尿毒综合征
Pediatr Nephrol. 2013 Aug;28(8):1315-8. doi: 10.1007/s00467-013-2492-x. Epub 2013 Apr 28.
2
Eculizumab in an anephric patient with atypical haemolytic uraemic syndrome and advanced vascular lesions.依库珠单抗治疗一名患有非典型溶血性尿毒症综合征和晚期血管病变的无肾患者。
Nephrol Dial Transplant. 2013 Nov;28(11):2899-907. doi: 10.1093/ndt/gft340. Epub 2013 Sep 5.
3
Clinical grand rounds: atypical hemolytic uremic syndrome.临床大查房：非典型溶血尿毒综合征。
Am J Nephrol. 2012;35(5):394-400. doi: 10.1159/000337954. Epub 2012 Apr 18.
4
Complement therapy in atypical haemolytic uraemic syndrome (aHUS).补体治疗非典型溶血尿毒症综合征（aHUS）。
Mol Immunol. 2013 Dec 15;56(3):199-212. doi: 10.1016/j.molimm.2013.05.224. Epub 2013 Jun 28.
5
Rare genetic variant in the CFB gene presenting as atypical hemolytic uremic syndrome and immune complex diffuse membranoproliferative glomerulonephritis, with crescents, successfully treated with eculizumab.补体因子B（CFB）基因中的罕见遗传变异表现为非典型溶血尿毒综合征和免疫复合物弥漫性膜增生性肾小球肾炎，伴有新月体形成，使用依库珠单抗治疗成功。
Pediatr Nephrol. 2017 May;32(5):885-891. doi: 10.1007/s00467-016-3577-0. Epub 2017 Feb 16.
6
Tailored eculizumab therapy in the management of complement factor H-mediated atypical hemolytic uremic syndrome in an adult kidney transplant recipient: a case report.成人肾移植受者中补体因子H介导的非典型溶血性尿毒症综合征管理中的个性化依库珠单抗治疗：一例报告
Transplant Proc. 2012 Dec;44(10):3037-40. doi: 10.1016/j.transproceed.2012.07.141.
7
A new era in the diagnosis and treatment of atypical haemolytic uraemic syndrome.非典型溶血尿毒综合征诊断与治疗的新时代。
Neth J Med. 2012 Apr;70(3):121-9.
8
Eculizumab as first-line therapy for atypical hemolytic uremic syndrome.依库珠单抗作为一线治疗用于非典型溶血性尿毒症综合征。
Pediatrics. 2014 Jun;133(6):e1759-63. doi: 10.1542/peds.2013-1787.
9
Cryptic activity of atypical hemolytic uremic syndrome and eculizumab treatment.非典型溶血尿毒综合征的隐匿性活动和依库珠单抗治疗。
Pediatrics. 2014 Jun;133(6):e1769-71. doi: 10.1542/peds.2013-2921. Epub 2014 May 19.
10
Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings.依库珠单抗治疗非典型溶血尿毒综合征：长期临床病程和组织学发现。
Pediatr Nephrol. 2011 Nov;26(11):2085-8. doi: 10.1007/s00467-011-1989-4. Epub 2011 Aug 30.

引用本文的文献

1
The Inhibitory Effects of a Factor B-Binding DNA Aptamer Family Supersede the Gain of Function of Factor B Variants Associated with Atypical Hemolytic Uremic Syndrome.Factor B 结合 DNA 适体家族的抑制作用超过与非典型溶血尿毒症综合征相关的因子 B 变体的功能获得。
J Immunol. 2024 Dec 1;213(11):1691-1702. doi: 10.4049/jimmunol.2400420.
2
Clinical Relapses of Atypical HUS on Eculizumab: Clinical Gap for Monitoring and Individualised Therapy.接受依库珠单抗治疗的非典型溶血尿毒综合征的临床复发：监测和个体化治疗的临床差距
Case Rep Nephrol. 2018 Feb 6;2018:2781789. doi: 10.1155/2018/2781789. eCollection 2018.
3
Critical appraisal of eculizumab for atypical hemolytic uremic syndrome.

本文引用的文献

1
Peripheral gangrene in children with atypical hemolytic uremic syndrome.儿童非典型溶血尿毒综合征的周围性坏疽。
Pediatrics. 2013 Jan;131(1):e331-5. doi: 10.1542/peds.2012-0903. Epub 2012 Dec 10.
2
Profound neurological injury in a patient with atypical hemolytic uremic syndrome.
Ann Hematol. 2013 Apr;92(4):557-8. doi: 10.1007/s00277-012-1615-y. Epub 2012 Nov 9.
3
Manipulating the mediator: modulation of the alternative complement pathway C3 convertase in health, disease and therapy.调控中介物：健康、疾病和治疗中替代补体途径 C3 转化酶的调节。
依库珠单抗治疗非典型溶血性尿毒症综合征的批判性评价。
J Blood Med. 2016 Apr 12;7:39-72. doi: 10.2147/JBM.S36249. eCollection 2016.
4
Diabetic ketoacidosis presenting with atypical hemolytic uremic syndrome associated with a variant of complement factor B in an adult: a case report.成人糖尿病酮症酸中毒合并非典型溶血尿毒综合征及补体因子B变异体：一例报告
J Med Case Rep. 2016 Feb 24;10:38. doi: 10.1186/s13256-016-0825-7.
5
Advances and challenges in the management of complement-mediated thrombotic microangiopathies.补体介导的血栓性微血管病管理中的进展与挑战
Ther Adv Hematol. 2015 Aug;6(4):171-85. doi: 10.1177/2040620715577613.
6
An international consensus approach to the management of atypical hemolytic uremic syndrome in children.儿童非典型溶血性尿毒症综合征管理的国际共识方法。
Pediatr Nephrol. 2016 Jan;31(1):15-39. doi: 10.1007/s00467-015-3076-8. Epub 2015 Apr 11.
7
Neonatal atypical hemolytic uremic syndrome from a factor H mutation treated with eculizumab.用依库珠单抗治疗的由因子H突变引起的新生儿非典型溶血性尿毒症综合征。
Clin Nephrol. 2015 Sep;84(3):181-5. doi: 10.5414/CN108532.
8
A complicated case of atypical hemolytic uremic syndrome with frequent relapses under eculizumab.一例在接受依库珠单抗治疗下频繁复发的非典型溶血性尿毒症综合征复杂病例。
Pediatr Nephrol. 2015 Jun;30(6):1039-42. doi: 10.1007/s00467-015-3078-6. Epub 2015 Mar 10.
9
Eculizumab therapy leads to rapid resolution of thrombocytopenia in atypical hemolytic uremic syndrome.依库珠单抗治疗可使非典型溶血性尿毒症综合征的血小板减少迅速得到缓解。
Adv Hematol. 2014;2014:295323. doi: 10.1155/2014/295323. Epub 2014 Oct 22.
10
Complement factor B mutations in atypical hemolytic uremic syndrome-disease-relevant or benign?非典型溶血性尿毒症综合征中补体因子B突变——与疾病相关还是良性？
J Am Soc Nephrol. 2014 Sep;25(9):2053-65. doi: 10.1681/ASN.2013070796. Epub 2014 Mar 20.
Immunobiology. 2012 Nov;217(11):1057-66. doi: 10.1016/j.imbio.2012.07.016.
4
Neonatal onset atypical hemolytic uremic syndrome successfully treated with eculizumab.新生儿起病的非典型溶血尿毒综合征经依库珠单抗治疗后获得成功。
Pediatr Nephrol. 2013 Jan;28(1):155-8. doi: 10.1007/s00467-012-2296-4. Epub 2012 Sep 6.
5
Eculizumab therapy in a child with hemolytic uremic syndrome and CFI mutation.依库珠单抗治疗伴有溶血尿毒综合征和 CFI 突变的患儿。
Pediatr Nephrol. 2012 Dec;27(12):2327-31. doi: 10.1007/s00467-012-2283-9. Epub 2012 Aug 19.
6
Atypical hemolytic uremic syndrome.非典型溶血尿毒综合征。
Orphanet J Rare Dis. 2011 Sep 8;6:60. doi: 10.1186/1750-1172-6-60.
7
Eculizumab: safety and efficacy after 17 months of treatment in a renal transplant patient with recurrent atypical hemolytic-uremic syndrome: case report.依库珠单抗治疗肾移植后复发非典型溶血尿毒综合征患者17个月的安全性和疗效：病例报告
Transplant Proc. 2010 Dec;42(10):4353-5. doi: 10.1016/j.transproceed.2010.09.125.
8
Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation.依库珠单抗诱导与 C3 基因突变相关的移植后复发性 HUS 长期缓解。
Pediatr Nephrol. 2011 Apr;26(4):613-9. doi: 10.1007/s00467-010-1708-6. Epub 2010 Dec 2.
9
Mutations in alternative pathway complement proteins in American patients with atypical hemolytic uremic syndrome.美国非典型溶血尿毒综合征患者旁路途径补体蛋白的突变。
Hum Mutat. 2010 Jun;31(6):E1445-60. doi: 10.1002/humu.21256.
10
The tick-over theory revisited: formation and regulation of the soluble alternative complement C3 convertase (C3(H2O)Bb).重温周转理论：可溶性替代补体C3转化酶（C3(H2O)Bb）的形成与调节
Mol Immunol. 2008 Apr;45(8):2370-9. doi: 10.1016/j.molimm.2007.11.003. Epub 2007 Dec 21.