非典型溶血性尿毒症治疗的研究进展  

Research Progress in the Cure of Atypical Hemolytic Uremic Syndrome

作　　者：戴艺萍 阮一平[2] 洪富源[2] DAI Yi-ping;RUAN Yi-ping;HONG Fu-yuan(Department of Nephrology,Shengli Clinical Medical College of Fujian Medical University,Fuzhou 350001,Fujian Province,China;Department of Nephrology,Fujian Provincial Hospital,Fuzhou 350001,Fujian Province,China)

机构地区：[1]福建医科大学省立临床医学院肾内科,福建福州350001 [2]福建省立医院肾内科,福建福州350001

出　　处：《罕少疾病杂志》2022年第7期1-4,共4页Journal of Rare and Uncommon Diseases

摘　　要：非典型溶血性尿毒综合征(aHUS)是一种由补体失调引起的血栓性微血管病(TMA),其表现为微血管病性溶血性贫血、血小板减少和终末器官损害(主要是肾脏)三联征。它发生于各个年龄段,以儿童期发病为主,其次为妊娠引起发病。其预后不良,死亡率高,属于罕见病中的疑难问题。长期以来,aHUS的诊断与治疗困扰着人们,这也促进了医学人士对此病的研究,而近年来C5补体抑制剂的诞生,为aHUS的治疗提供了更多可能性,已经逐渐成为一线推荐治疗,但由于其成本高,多数患者仍以选择血浆置换为主。2019年新批准用于治疗aHUS的长效C5补体抑制剂雷夫利珠单抗(ravulizumab)也被证实比短效C5补体抑制剂依库珠单抗(eculizumab)更方便、总治疗费用更少,且没有脑膜炎球菌感染等重大不良反应。也有研究表明对于难治性aHUS,血浆置换后,在激素使用基础上,利妥昔单抗(rituximab)联合他克莫司(tacrolimus),可能是一种可行的疗法。因此,本文主要针对治疗aHUS的补体调节疗法、免疫抑制疗法的研究进展进行概述。Atypical hemolytic uremic syndrome(aHUS)is a thrombotic microangiopathy(TMA)caused by complement imbalance.It is characterized by a triad of microvascular hemolytic anemia,thrombocytopenia and end organs(mainly kidney)damage.It occurs at all ages,mainly in childhood,secondly in pregnancy.With a poor prognosis and high mortality,it is a difficult problem in rare diseases.For a long time,the diagnosis and treatment of aHUS have plagued human,but also promoted the research on this disease.In recent years,the birth of C5 complement inhibitor has provided more possibilities for the aHUS patients,and gradually become the first-line recommended treatment.However,due to its high cost,most patients still choose plasma exchange.Ravulizumab,a long-acting C5 complement inhibitor newly approved for the treatment of aHUS in 2019,with more convenient,less cost,no risk of Neisseria meningitidis infection and other major adverse reactions,has also been proved to be better than eculizumab,a short-acting C5 complement inhibitor.Some studies have also expounded that rituximab combined tacrolimus may be a feasible treatment for refractory aHUS on the basis of using hormone after plasma exchange.Therefore,this paper mainly reviews the research progress of complement regulation therapy and immunosuppressive therapy for aHUS.

关 键 词：溶血性尿毒综合征 依库珠单抗 雷夫利珠单抗 利妥昔单抗 疗法 

分 类 号：R692.5[医药卫生—泌尿科学]