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补体因子I突变导致的非典型溶血尿毒综合征

Atypical hemolytic-uremic syndrome due to complement factor I mutation.

作者信息

Almalki Abdullah H, Sadagah Laila F, Qureshi Mohammed, Maghrabi Hatim, Algain Abdulrahman, Alsaeed Ahmed

机构信息

Ministry of National Guard, Jeddah 21423, Saudi Arabia.

出版信息

World J Nephrol. 2017 Nov 6;6(6):243-250. doi: 10.5527/wjn.v6.i6.243.

Abstract

Atypical hemolytic-uremic syndrome (aHUS) is a rare disease of complement dysregulation leading to thrombotic microangiopathy (TMA). Renal involvement and progression to end-stage renal disease are common in untreated patients. We report a 52-year-old female patient who presented with severe acute kidney injury, microangiopathic hemolytic anemia, and thrombocytopenia. She was managed with steroid, plasma exchange, and dialysis. Kidney biopsy shows TMA and renal cortical necrosis. Genetic analysis reveals heterozygous complement factor I (CFI) mutation. Eculizumab was initiated after 3 mo of presentation, continued for 9 mo, and stopped because of sustained hematologic remission, steady renal function, and cost issues. Despite this, the patient continued to be in hematologic remission and showed signs of renal recovery, and peritoneal dialysis was stopped 32 mo after initiation. We report a case of aHUS due to CFI mutation, which, to the best of our knowledge, has not been reported before in Saudi Arabia. Our case illustrates the challenges related to the diagnosis and management of this condition, in which a high index of suspicion and prompt treatment are usually necessary.

摘要

非典型溶血尿毒综合征(aHUS)是一种罕见的补体调节异常疾病,可导致血栓性微血管病(TMA)。未经治疗的患者常见肾脏受累并进展至终末期肾病。我们报告了一名52岁女性患者,她表现为严重急性肾损伤、微血管病性溶血性贫血和血小板减少。她接受了类固醇、血浆置换和透析治疗。肾活检显示TMA和肾皮质坏死。基因分析显示杂合性补体因子I(CFI)突变。在出现症状3个月后开始使用依库珠单抗,持续使用9个月,因血液学持续缓解、肾功能稳定及费用问题停药。尽管如此,患者仍处于血液学缓解状态并显示出肾脏恢复的迹象,开始腹膜透析32个月后停止。我们报告了一例由CFI突变引起的aHUS病例,据我们所知,此前在沙特阿拉伯尚未有过报道。我们的病例说明了这种疾病在诊断和管理方面的挑战,通常需要高度的怀疑指数和及时的治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0a89/5714872/bef3aef3304b/WJN-6-243-g001.jpg

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