Atypical hemolytic uremic syndrome
Atypical hemolytic uremic syndrome
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DOI：
10.1182/asheducation-2016.1.217
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发表时间：
2016-12-01
影响因子：
3
通讯作者：
Afshar-Kharghan, Vahid
中科院分区：
文献类型：
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作者：
Afshar-Kharghan, Vahid
非典型溶血性尿毒综合征（阿胡斯）是一种血栓性微血管病（TMA），其影响多个器官，特别是肾脏。阿胡斯可以是散发性的或家族性的，并且最常见的是由旁路补体途径的失调引起的。阿胡斯的初始发作可发生在任何年龄，并且与进展为终末期肾病的高比率相关。许多阿胡斯患者在原生或移植肾中复发，需要密切监测和长期管理。抗补体治疗的可用性已经彻底改变了阿胡斯的管理，并且可以通过诱导血液学缓解、改善或稳定肾功能以及预防移植物衰竭来改变阿胡斯的自然病程。因此，重要的是成功地完成区分阿胡斯与其他TMA的挑战性任务，并在疾病过程中早期开始适当的治疗。考虑到目前可用的抗补体疗法的高成本，从财务角度来看，在疾病过程中早期准确诊断阿胡斯并确定必要的治疗长度也是重要的。这突出了开发具有快速周转时间的精确补体功能和遗传研究的需要。
Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) that affects multiple organs and the kidneys in particular. aHUS can be sporadic or familial and is most commonly caused by dysregulation of the alternative complement pathway. The initial attack of aHUS can occur at any age, and is associated with a high rate of progression to end stage renal disease. Many aHUS patients relapse in the native or transplanted kidneys, and require close monitoring and long-term management. Availability of anticomplement therapy has revolutionized the management of aHUS, and can change the natural course of aHUS by inducing hematologic remission, improving or stabilizing kidney functions, and preventing graft failure. As a result, it is important to succeed in the challenging task of differentiating aHUS from other TMAs and initiate adequate treatment early during the course of disease. Considering the high cost of currently available anticomplement therapy, it is important also from a financial point of view to accurately diagnose aHUS early during the course of disease and determine the necessary length of therapy. This highlights the need for development of precise complement functional and genetic studies with rapid turnaround time.