中国儿童非典型溶血尿毒综合征诊治专家共识(2023版)  

Consensus on the diagnosis and treatment of atypical hemolytic uremic syndrome in Chinese children(2023 edition)

作　　者：中国罕见病联盟儿童非典型溶血尿毒综合征专业委员会 国家儿童医学中心(首都医科大学附属北京儿童医院) 《中华实用儿科临床杂志》编辑委员会 倪鑫[2] 刘小荣[3] 陈植[3] 樊剑锋[3] China Alliance for Rare Diseases Pediatric Atypical Hemolytic Uremic Syndrome Committee;National Center for Children′s Health(Beijing Children′s Hospital,Capital Medical University);Editorial Board of Chinese Journal of Applied Clinical Pediatrics

机构地区：[1]不详 [2]国家儿童医学中心、首都医科大学附属北京儿童医院 [3]首都医科大学附属北京儿童医院

出　　处：《中华实用儿科临床杂志》2023年第6期401-412,共12页Chinese Journal of Applied Clinical Pediatrics

基　　金：北京市科学技术委员会首都临床特色应用研究与成果推广(Z161100000516106);北京市科学技术委员会重点项目(D181100000118006);首都卫生发展科研专项项目(2016-2-2094)。

摘　　要：非典型溶血尿毒综合征(atypical hemolytic uremic syndrome,aHUS)是罕见病和危重性疾病,由补体旁路调节蛋白异常所致。急性期病死率高,且病情反复,容易进展为终末期肾病。近年来,随着补体抑制剂依库珠单抗的应用,aHUS患者的预后有了较大改善。我国aHUS诊治水平在不同地区差异较大,临床医师对aHUS认识不足,会导致疾病诊断及治疗延迟。为进一步规范我国儿童aHUS的诊断、治疗和管理,由中国罕见病联盟儿童非典型溶血尿毒综合征专业委员会牵头,结合我国病例特点及临床经验,组织国内专家制定本共识。Atypical hemolytic uremic syndrome(aHUS)is a rare and life-threatening disease caused by dysregulation of the complement alternative pathway,with a high mortality in the acute phase.The condition is prone to recurrent and to progression to end-stage kidney disease.With the application of the humanized monoclonal antibody that blocks terminal complement C5 activation in recent years,the prognosis of aHUS has improved considerably.There are problems of large differences in the diagnostic and therapeutic level in difference regions,insufficient recognition of clinicians,and delayed diagnosis as well as treatment of aHUS in China.In order to standardize the diagnosis,treatment,and management of aHUS in Chinese children,the China Alliance for Rare Diseases Pediatric Atypical Hemolytic Uremic Syndrome Committee took the lead and organized domestic experts to compile the expert consensus,combined with the characteristics and clinical experience of Chinese children.

关 键 词：儿童 溶血尿毒综合征 非典型 诊断 治疗 共识 

分 类 号：R726.9[医药卫生—儿科]