非典型溶血尿毒综合征
发病机制
医学
替代补体途径
封锁
补体系统
免疫学
伊库利珠单抗
补语(音乐)
透析
重症监护医学
内科学
抗体
表型
受体
生物
遗传学
基因
互补
作者
Juliette Léon,Marie‐Bénédicte LeStang,Rébecca Sberro‐Soussan,Aude Servais,Dany Anglicheau,Véronique Frémeaux‐Bacchi,Julien Zuber
摘要
Abstract Overactivation of the complement alternative pathway drives the pathogenesis of primary atypical hemolytic uremic syndrome (aHUS). Genetically‐determined or acquired dysregulation of the complement is frequently identified in patients with aHUS, pregnancy‐related hemolytic uremic syndrome (HUS), and severe hypertension‐associated HUS. In contrast, it is still unclear whether self‐limited complement activation, which frequently occurs in other forms of HUS, provides key mechanistic clues or results from endothelial damage. Development of novel biomarkers is underway to firmly establish complement‐driven pathogenesis. C5 blockade therapy has revolutionized the management of aHUS patients, resulting in a halving of the subpopulation under chronic dialysis over the course of a few years. On the other hand, the efficacy of C5 blockade in secondary forms of HUS, as assessed by small and uncontrolled case series, is less compelling and should be investigated through properly designed prospective clinical trials. The increased risk of meningococcal infection, related to C5 inhibition, must be rigorously addressed with suitable prophylaxis. Treatment duration should be determined based on an individualized benefit/risk assessment.
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