Update on hemolytic uremic syndrome:Diagnostic and therapeutic recommendations

作     者：Maurizio Salvadori Elisabetta Bertoni 

作者机构：Renal UnitCareggi HospitalCareggi UniversityFlorence 50139Italy 

出 版 物：《World Journal of Nephrology》 (世界肾病学杂志（英文版）)

年 卷 期：2013年第2卷第3期

页      面：56-76页

学科分类：1002[医学-临床医学] 100210[医学-外科学(含：普外、骨外、泌尿外、胸心外、神外、整形、烧伤、野战外)] 10[医学] 

主　　题：Enterohemorrhagic Escherichia coli Diarrhea, Shiga toxin (Stx) 1 and Stx 2 Atypical hemolytic uremic syndrome Complement factors Thrombotic thrombocytopenic purpura Secondary thrombotic microangiopathy Plasma therapy Eculizumab 

摘      要：Hemolytic uremic syndrome （HUS） is a rare disease. In this work the authors review the recent findings on HUS, considering the different etiologic and patho-genetic classifications. New findings in genetics and, in particular, mutations of genes that encode the complement-regulatory proteins have improved our understanding of atypical HUS. Similarly, the comple-ment proteins are clearly involved in all types of thrombotic microangiopathy： typical HUS, atypical HUS and thrombotic thrombocytopenic purpura （TTP）. Fur-thermore, several secondary HUS appear to be related to abnormalities in complement genes in predisposed patients. The authors highlight the therapeutic as-pects of this rare disease, examining both “traditional therapy （including plasma therapy, kidney and kidney-liver transplantation） and “new therapies. The latter include anti-Shiga-toxin antibodies and anti-C5 mono-clonal antibody “eculizumab. Eculizumab has been recently launched for the treatment of the atypical HUS, but it appears to be effective in the treatment of typical HUS and in TTP. Future therapies are in phases Ⅰ and Ⅱ. They include anti-C5 antibodies, which are more purifed, less immunogenic and absorbed orally and, anti-C3 antibodies, which are more powerful, but potentially less safe. Additionally, infusions of recombinant complement-regulatory proteins are a potential future therapy.