University Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Pediatrics, AK Hamburg Nord, Asklepios Medical School, Hamburg, Germany.
Pediatr Nephrol. 2018 Aug;33(8):1277-1281. doi: 10.1007/s00467-018-3972-9. Epub 2018 May 17.
Hemolytic uremic syndrome caused by Shiga toxin-producing E. coli (STEC-HUS) is often associated with a severe morbidity including neurological involvement and a mortality of 1-5%. Although STEC-HUS is often self-limited, improvement of treatment strategies is needed for cases with complications and, among others, plasma exchange/plasmapheresis and use of antibiotics have been advocated. With the availability of the complement blocker eculizumab, now a standard treatment of atypical HUS, several series have addressed its use in STEC-HUS, with variable response; randomized controlled trials are lacking.In this issue of Pediatric Nephrology, Pecheron et al. present a cohort of 33 pediatric patients with severe HUS treated with eculizumab. Neurological involvement was observed in 85% of the patients and 94% required dialysis. Most patients (55%) did not benefit from eculizumab and renal dysfunction as well as neurological sequelae did not resolve. In a subgroup of patients, however, rapid neurological improvement was described. In the post-hoc-defined group of patients with favorable outcome, there was a trend towards more sustained complement inhibition, although this finding was not significant compared to patients with an unfavorable outcome.Because multiple interventions were used and the study did not include any control group, future controlled studies are urgently needed to resolve the debate as to whether eculizumab can be an effective treatment for both prevention and treatment of complications in STEC-HUS.
产志贺毒素大肠杆菌（STEC-HUS）引起的溶血性尿毒症综合征常伴有严重的发病率，包括神经系统受累和 1-5%的死亡率。尽管 STEC-HUS 通常是自限性的，但需要改进治疗策略，以应对伴有并发症的病例，其中包括血浆置换/血浆滤过和使用抗生素等方法。由于补体抑制剂依库珠单抗的出现，现在已经成为非典型 HUS 的标准治疗方法，已有多个系列研究探讨了其在 STEC-HUS 中的应用，反应不一；但目前缺乏随机对照试验。在本期《儿科肾脏病学》中，Pecheron 等人报告了一组 33 例接受依库珠单抗治疗的严重 HUS 儿科患者的队列研究。85%的患者存在神经系统受累，94%需要透析。大多数患者（55%）未从依库珠单抗中获益，肾功能障碍和神经系统后遗症均未得到缓解。然而，在后述定义的具有良好结局的亚组患者中，描述了快速的神经改善。在具有良好结局的患者中，补体抑制更持久，尽管与预后不良的患者相比，这一发现没有显著差异，但这一发现也没有显著差异。由于采用了多种干预措施，且该研究未包括任何对照组，因此迫切需要进行未来的对照研究，以解决依库珠单抗是否能有效预防和治疗 STEC-HUS 并发症的争议。
