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    Renal recovery with eculizumab in atypical hemolytic uremic syndrome following prolonged dialysis

    发布于：2013年4月22日    文字：【大】【中】【小】

    Clin Nephrol. 2013 Apr 5. [Epub ahead of print]

    Renal recovery with eculizumab in atypical hemolytic uremic syndrome following prolonged dialysis.

    Povey H, Vundru R, Junglee N, Jibani M.

    Abstract

    Hemolytic uremic syndrome (HUS) is a thrombotic microangiopathy (TMA) which encompasses hemolytic anemia, thrombocytopenia, and organ impairment.Around 10% of cases are atypical HUS (aHUS), a rare disease with poor outcomes caused by uncontrolled activation of the alternative complement pathway. This case describes a young woman with clinical manifestations compatible with TMA during childhood and adolescence who was formally diagnosed with aHUS at the age of 21. She was managed with intensive plasma exchange and hemodialysis, which failed to improve her severe acute kidney injury and other hematological manifestations of aHUS. This was further compounded by several episodes of flash pulmonary edema and the posterior reversible encephalopathy syndrome (PRES).Treatment with the monoclonal anti-C5 inhibitor, eculizumab, improved all hematological parameters with almost full renal recovery following 3.5 months of dialysis. So far, long-term use of eculizumab (> 11 months) continues to be effective and without complication. Our case illustrates the difficulty but importance of early consideration of aHUS in patients presenting with TMA. More importantly, we highlight that near-normal renal recovery may be attained with eculizumab in adults even after a long dependence on dialysis - an observation that has not been reported in the literature so far.

    依库利单抗治疗后使长期血透的aHUS患者肾功恢复

    溶血尿毒综合征(HUS)是一种血栓性微血管病(TMA)，临床症状包括溶血性贫血、血小板减低和器官损害。HUS中大约有10%的患者为不典型HUS(aHUS)。aHUS是一种由补体旁路过度激活引起的罕见病，患者预后较差。本病例为一个青年女性，在幼年和青春期时曾表现为类似aHUS的临床症状，在21岁时被确诊为aHUS。患者住院后进行了强效血浆置换和血液透析治疗，但急性肾损害和其他血液系统症状并未好转。患者同时出现了一过性肺水肿和后部可逆性脑病综合征。在使用抗C5单克隆抗体(依库利单抗)治疗后，患者所有血液系统症状均好转。后经过3.5个月的血液透析治疗，患者的肾功能也几乎完全恢复。至今，长疗程(>11个月)的依库利单抗治疗依然对该患者有效，患者并未出现并发症。本病例说明了早期诊断aHUS的困难性和重要性。更重要的是，对于成人aHUS，即使已经进行了长时间的血液透析治疗，依库利单抗的应用仍然有可能逆转患者的肾功能，这个发现是以往文献中没有报道过的。