Department of Pediatric Nephrology, Radboud University Medical Center Amalia Children's Hospital, Nijmegen, The Netherlands.
Department of Nephrology, Radboud University Medical Center, Nijmegen, The Netherlands.
Nephrol Dial Transplant. 2018 Apr 1;33(4):635-645. doi: 10.1093/ndt/gfx196.
Atypical haemolytic uremic syndrome (aHUS) is a rare but severe form of thrombotic microangiopathy as a consequence of complement dysregulation. aHUS has a poor outcome with high mortality and >50% of patients developing end-stage renal disease. Since the end of 2012, these outcomes have greatly improved with the introduction of eculizumab. Currently the duration of treatment is debated. Most guidelines advise lifelong treatment. However, there is no hard evidence to support this advice. Historically, a substantial number of aHUS patients were weaned of plasma therapy, often without disease recurrence. Moreover, the long-term consequences of eculizumab treatment are unknown. In this retrospective study we describe 20 patients who received a restrictive treatment regimen.
All aHUS patients who presented in the Radboud University Medical Center, Nijmegen, The Netherlands, between 2012 and 2016 and who received eculizumab are described. Clinical, diagnostic and follow-up data were gathered and reviewed.
Twenty patients (14 adults, 6 children) with aHUS have received eculizumab. Eculizumab was tapered in all and stopped in 17 patients. aHUS recurrence occurred in five patients. Due to close monitoring, recurrence was detected early and eculizumab was restarted. No clinical sequela such as proteinuria or progressive kidney dysfunction was detected subsequently. In total, eculizumab has been discontinued in 13 patients without aHUS recurrence, of which 5 are event free for >1 year. With this strategy ∼€11.4 million have been saved.
A restrictive eculizumab regimen in aHUS appears safe and effective. Prospective studies should further evaluate the most optimal treatment strategy.
非典型溶血性尿毒症综合征（aHUS）是补体失调导致的血栓性微血管病的一种罕见但严重的形式。由于补体调节异常，aHUS 结局较差，死亡率高，超过 50%的患者发展为终末期肾病。自 2012 年底以来，随着依库珠单抗的引入，这些结果得到了极大改善。目前，治疗持续时间存在争议。大多数指南建议终身治疗。然而，目前没有确凿的证据支持这一建议。从历史上看，大量的 aHUS 患者可以逐渐减少血浆治疗，且通常不会出现疾病复发。此外，依库珠单抗治疗的长期后果尚不清楚。在这项回顾性研究中，我们描述了接受限制治疗方案的 20 名患者。
描述了 2012 年至 2016 年间在荷兰奈梅亨拉德堡德大学医学中心就诊并接受依库珠单抗治疗的所有 aHUS 患者。收集并回顾了临床、诊断和随访数据。
20 名（14 名成人，6 名儿童）aHUS 患者接受了依库珠单抗治疗。所有患者均逐渐减少依库珠单抗剂量并停药。有 5 名患者出现 aHUS 复发。由于密切监测，早期发现了复发并重新开始使用依库珠单抗。随后未发现蛋白尿或进行性肾功能障碍等临床后遗症。依库珠单抗共停用 13 例，未复发，其中 5 例无事件发生>1 年。通过这种策略，节省了约 1140 万欧元。
在 aHUS 中采用限制依库珠单抗方案似乎是安全有效的。应进一步进行前瞻性研究，以评估最优化的治疗策略。
