依库珠单抗治疗非典型溶血尿毒综合征的疗效  

Eculizumab treatment for atypical haemolytic uremic syndrome

作　　者：张炎炎 王蔚[2] 李贵森[2] 陈莎莎 ZHANG Yanyan;WANG Wei;LI Guisen;CHEN Shasha(School of Medicine,University of Electronic Science and Technology of China,Chengdu 610054,China;Department of Nephrology,Sichuan Academy of Medical Sciences&Sichuan Provincial People s Hospital,Chengdu 610072,China)

机构地区：[1]电子科技大学医学院,成都610054 [2]四川省医学科学院·四川省人民医院肾脏内科

出　　处：《肾脏病与透析肾移植杂志》2024年第4期308-314,共7页Chinese Journal of Nephrology,Dialysis & Transplantation

摘　　要：目的:分析5例非典型溶血尿毒综合征(aHUS)患者短期使用依库珠单抗的疗效及不良反应。方法:回顾性分析2023年1月至2024年5月在四川省人民医院规律治疗的5例aHUS患者资料,收集患者实验室检查、肾脏病理检查、基因检测结果,依库珠单抗治疗效果和不良反应。结果:5例aHUS患者中4例为女性,中位年龄40(17~66)岁,其中2例为原发性aHUS,肾移植相关、恶性高血压和系统性红斑狼疮各1例。临床均表现为急性肾损伤、蛋白尿、血尿、贫血、血小板减少,2例补体C3下降。3例肾活检病理示内皮细胞损伤、微血栓形成。3例接受透析,4例患者使用激素治疗(2例接受大剂量激素冲击),肾移植受者接受兔抗人胸腺免疫球蛋白和静脉丙种球蛋白治疗,1例系统性红斑狼疮患者接受CD20单抗治疗。从诊断aHUS到接受依库珠单抗治疗的中位时间为20(2~37.5)d,依库珠单抗治疗4~20次,治疗后平均7(4.5~8.5)d所有患者均达到血液学缓解,2例患者获得肾脏缓解,1例患者脱离透析。随访8~24周血清肌酐、乳酸脱氢酶均较治疗前显著降低,估算的肾小球滤过率、血红蛋白、血小板较治疗前上升(P<0.001),未见明显不良反应。结论:aHUS临床表现多样,进展快,预后差,一旦确诊立即启动依库珠单抗治疗,可快速改善患者的血液学指标和肾功能,长期临床获益仍需进一步观察随访。Objective:To analyse efficacy of short-term use of complement C5 inhibitor eculizumab,and adverse reactions in patients with atypical haemolytic uremic syndrome(aHUS).Methodology:Five patients with aHUS who were regularly treated in Sichuan Provincial People s Hospital between January 2023 and May 2024 were retrospectively collected and analysed for clinical manifestations,laboratory examinations,renal pathological examinations,genetic test results,the therapeutic efficacy of eculizumab and the adverse reactions.Results:One of the five patients with aHUS was male,with a median age of 40(17~66)years,etiologu included renal transplantation,malignant hypertension,autoimmune disease,and primary aHUS.All presented clinically with acute renal failure,proteinuria,haematuria,anaemia and thrombocytopenia,dicrease of complement C3.Renal biopsy was performed in 3 cases and showed endothelial cell damage and microthrombosis.3 cases received dialysis,3 cases had renal biopsies,all 4 patients were treated with steroids(2 received methylprednisolone pulse therapy),1 kidney transplant-related case was treated with rabbit anti-human thymus immunoglobulin(ATG),intravenous gammaglobulin,and 1 with CD20 monoclonal antibody(with systemic lupus erythematosus in the primary disease).The median time from diagnosis of aHUS to treatment with eculizumab was 20(2~37.5)days,with 4~20 times of eculizumab,and a mean of 7(4.5~8.5)days post-treatment all patients achieved haematological remission,3 patients achieved nephrological remission,and 1 patient was taken off dialysis.At 8~24 weeks months of follow-up creatinine and LDH were significantly lower and eGFR,haemoglobin and platelets were significantly higher than before treatment(P<0.001).No significant adverse reactions were found.Conclusion:aHUS has diverse clinical manifestations,rapid progression,and poor prognosis,and eculizumab is the first-line treatment option for aHUS.Once diagnosed,treatment can be initiated immediately,which can rapidly improve the haematological indexes and renal

关 键 词：非典型溶血尿毒综合征 依库珠单抗 不良反应 

分 类 号：R692[医药卫生—泌尿科学]