Department of Paediatric Nephrology, Evelina Children's Hospital, London, UK.
Clin Kidney J. 2012 Feb;5(1):34-6. doi: 10.1093/ndtplus/sfr174. Epub 2012 Jan 30.
Disorders in complement regulation are a major cause of atypical haemolytic-uraemic syndrome (aHUS). Eculizumab, a monoclonal antibody targeting complement C5 and blocking the terminal complement cascade, should theoretically be useful in this disease, particularly when associated with specific complement pathway anomalies such as Factor H deficiency. Eculizumab is emerging as an effective treatment for post-transplant aHUS recurrence and may have a role in treating de novo aHUS, halting the haemolytic process. In this case report, we describe the fourth case of aHUS treated with eculizumab. In our patient, with a known complement Factor H mutation, not only has the disease process become quiescent but also this therapy has led to significantly improved renal function so that dialysis is no longer necessary.
补体调节紊乱是非典型溶血尿毒综合征(aHUS)的主要病因。依库珠单抗是一种针对补体 C5 的单克隆抗体,可阻断末端补体级联反应,理论上在这种疾病中应该是有用的,尤其是当与特定的补体途径异常(如因子 H 缺乏)相关时。依库珠单抗作为移植后 aHUS 复发的有效治疗方法正在出现,并且可能在治疗新发性 aHUS 方面发挥作用,从而阻止溶血性过程。在本病例报告中,我们描述了第四例接受依库珠单抗治疗的 aHUS 患者。在我们的患者中,已知存在补体因子 H 突变,不仅疾病过程已处于静止状态,而且这种治疗还导致肾功能显著改善,以至于不再需要透析。