Korean J Intern Med.  2020 Jan;35(1):25-40. 10.3904/kjim.2019.388.

Consensus regarding diagnosis and management of atypical hemolytic uremic syndrome

• Lee H
• Kang E
• Kang HG
• Kim YH
• Kim JS
• Kim HJ
• Moon KC
• Ban TH
• Oh SW
• Jo SK
• Cho H
• Choi BS
• Hong J
• Cheong HI
• Oh D

• KMID: 2501984
• DOI: http://doi.org/10.3904/kjim.2019.388

Abstract

Thrombotic microangiopathy (TMA) is defined by specific clinical characteristics, including microangiopathic hemolytic anemia, thrombocytopenia, and pathologic evidence of endothelial cell damage, as well as the resulting ischemic end-organ injuries. A variety of clinical scenarios have features of TMA, including infection, pregnancy, malignancy, autoimmune disease, and medications. These overlapping manifestations hamper differential diagnosis of the underlying pathogenesis, despite recent advances in understanding the mechanisms of several types of TMA syndrome. Atypical hemolytic uremic syndrome (aHUS) is caused by a genetic or acquired defect in regulation of the alternative complement pathway. It is important to consider the possibility of aHUS in all patients who exhibit TMA with triggering conditions because of the incomplete genetic penetrance of aHUS. Therapeutic strategies for aHUS are based on functional restoration of the complement system. Eculizumab, a monoclonal antibody against the terminal complement component 5 inhibitor, yields good outcomes that include prevention of organ damage and premature death. However, there remain unresolved challenges in terms of treatment duration, cost, and infectious complications. A consensus regarding diagnosis and management of TMA syndrome would enhance understanding of the disease and enable treatment decision-making.

Keyword

Thrombotic microangiopathies; Atypical hemolytic uremic syndrome; Complement pathway, alternative; Diagnosis, differential; Eculizumab