Rafat C, Coppo P, Fakhouri F, Frémeaux-Bacchi V, Loirat C, Zuber J, Rondeau E
Urgences néphrologiques et transplantation rénale, hôpital Tenon, Assistance publique-Hôpitaux de Paris, 75970 Paris, France; Centre national de référence des microangiopathies thrombotiques (CNR-MAT), AP-HP, 75012Paris, France.
Centre national de référence des microangiopathies thrombotiques (CNR-MAT), AP-HP, 75012Paris, France; Service d'hématologie, hôpital Saint-Antoine, 75970Paris, France; Unité Inserm UMR 1170, Assistance publique-Hôpitaux de Paris, 94805Villejuif, France.
Rev Med Interne. 2017 Dec;38(12):833-839. doi: 10.1016/j.revmed.2017.07.005. Epub 2017 Sep 22.
Major achievements in the understanding of thrombotic microangiopathies (TMA) have not only resulted in a reclassification of TMA but most of all they have culminated in the design of new treatments and have enabled clinicians to better delineate their prognosis. Recent multicenter studies have improved our understanding of the prognosis of atypical hemolytic and uremic syndromes (aHUS). More specifically, they have highlighted the role of genetic testing on predicting the recurrence of aHUS, the risk of chronic kidney disease and the recurrence following kidney transplantation. A major advance consisted of the identification of the alternative complement pathway in the pathogenesis of aHUS, thus paving the way for the use of the C5a inhibitor eculizumab in this indication. Eculizumab has thereafter dramatically improved the management of patients affected with aHUS. During spring 2011, a great epidemic of entero-hemorrhagic Escherichia coli (EHEC) associated HUS occurred in Germany, providing clinicians the opportunity to examine the relevance of antibiotic prophylaxis, plasma exchange and eculizumab in EHEC-associated HUS. In this work, we herein present advances achieved in the setting of therapeutic management and prognosis in HUS and other related TMA syndromes.
在血栓性微血管病（TMA）认识方面的重大成就不仅导致了TMA的重新分类，更重要的是，这些成就最终促成了新治疗方法的设计，并使临床医生能够更好地判断其预后。最近的多中心研究增进了我们对非典型溶血性尿毒症综合征（aHUS）预后的了解。更具体地说，这些研究突出了基因检测在预测aHUS复发、慢性肾脏病风险以及肾移植后复发方面的作用。一项重大进展是在aHUS发病机制中发现了替代补体途径，从而为在该适应症中使用C5a抑制剂依库珠单抗铺平了道路。此后，依库珠单抗极大地改善了aHUS患者的治疗。2011年春季，德国发生了一起与肠出血性大肠杆菌（EHEC）相关的溶血尿毒综合征大流行，为临床医生提供了检验抗生素预防、血浆置换和依库珠单抗在EHEC相关溶血尿毒综合征中的相关性的机会。在这项工作中，我们在此展示了在溶血尿毒综合征及其他相关TMA综合征的治疗管理和预后方面所取得的进展。
