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一例由股骨颈骨折引发的伴有补体因子I突变的非典型溶血性尿毒症综合征病例。

A Case of Atypical Hemolytic Uremic Syndrome With a Complement Factor I Mutation Triggered by a Femoral Neck Fracture.

作者信息

Kano Toshiki, Io Hiroaki, Sasaki Yu, Muto Masahiro, Muto Sayaka, Ogiwara Kei, Ikeda Arisa, Iwasaki Hiroyuki, Suzuki Yusuke

机构信息

Department of Nephrology, Juntendo University Nerima Hospital, Tokyo, Japan.

Department of Nephrology, Juntendo University Faculty of Medicine, Tokyo, Japan.

出版信息

Nephrology (Carlton). 2025 Mar;30(3):e70010. doi: 10.1111/nep.70010.

Abstract

Atypical hemolytic uremic syndrome is a thrombotic microangiopathy caused by the abnormal activation of the alternative complement pathway. Mutations in complement-related genes and autoantibodies against complement regulators are involved in the pathogenesis of this condition; the frequency of, and prognosis of patients harbouring, each genetic mutation varies based on the region and race. Complement factor I (CFI) mutations have been observed in 4%-8% of cases in Europe; however, they have not yet been reported in Japan. We present the first Japanese case of atypical hemolytic uremic syndrome in a patient harbouring a CFI mutation. An 83-year-old female patient presented with severe acute kidney injury, thrombocytopenia, and hemolytic anaemia following a femoral neck fracture. Plasma exchange and haemodialysis were initiated, resulting in improved kidney function and platelet count. However, the platelet count decreased when plasma exchange was discontinued. Therefore, we administered ravulizumab, an anti-complement 5 monoclonal antibody, which led to the maintenance of stable kidney function and platelet count. Genetic analysis revealed a CFI mutation, and the patient was treated with ravulizumab for 2 years without relapse. Individuals diagnosed with atypical hemolytic uremic syndrome harbouring CFI mutations experience poor outcomes, including low rates of remission, high rates of mortality, and progression to end-stage kidney disease. Our case serves as a crucial example demonstrating how prompt identification and appropriate management can lead to better patient outcomes.

摘要

非典型溶血尿毒综合征是一种由替代补体途径异常激活引起的血栓性微血管病。补体相关基因的突变和针对补体调节因子的自身抗体参与了这种疾病的发病机制;每种基因突变患者的发生率和预后因地区和种族而异。在欧洲,4%-8%的病例中观察到补体因子I(CFI)突变;然而,日本尚未有相关报道。我们报告了首例携带CFI突变的日本非典型溶血尿毒综合征病例。一名83岁女性患者在股骨颈骨折后出现严重急性肾损伤、血小板减少和溶血性贫血。开始进行血浆置换和血液透析,肾功能和血小板计数得到改善。然而,停止血浆置换后血小板计数下降。因此,我们给予抗补体5单克隆抗体ravulizumab,这使得肾功能和血小板计数得以维持稳定。基因分析发现了CFI突变,该患者接受ravulizumab治疗2年无复发。诊断为携带CFI突变的非典型溶血尿毒综合征患者预后较差,包括缓解率低、死亡率高以及进展为终末期肾病。我们的病例是一个关键例子,表明及时识别和适当管理可带来更好的患者预后。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/87c6/11861886/34811f61496d/NEP-30-0-g001.jpg

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