Recent insights into C3 glomerulopathy
Recent insights into C3 glomerulopathy
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DOI：
10.1093/ndt/gfs430
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发表时间：
2013-07-01
影响因子：
6.1
通讯作者：
Cook, H. Terence
中科院分区：
文献类型：
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作者：
Barbour, Thomas D.;Pickering, Matthew C.;Cook, H. Terence
“C3肾小球病”是一种最近的疾病分类，包括几种罕见类型的肾小球肾炎（GN），包括致密沉积病（DDD）、C3肾小球肾炎（C3GN）和CFHR5肾病。这些疾病都有一个关键的组织学特征，即肾小球内孤立的补体C3沉积。在过去的十年中，一种涉及补体替代途径（AP）失调的常见病因已经被阐明，在一定比例的患者中可以识别出遗传缺陷和/或自身抗体。我们回顾了C3肾小球病变的临床和组织学特征，并将其与潜在的分子机制联系起来。强调不受控制的C3激活在发病机制中的作用，并从动物模型中得到重要的教训。本文讨论了基因检测在C3肾小球病变个体或家族评估中的方法、优点和局限性。虽然还没有任何治疗方法显示出一贯有效，但针对补体系统特定成分的药物的临床评估正在进行中。然而，目前关于自然史和建议治疗的适当时间和持续时间的知识的局限性需要得到解决。
'C3 glomerulopathy' is a recent disease classification comprising several rare types of glomerulonephritis (GN), including dense deposit disease (DDD), C3 glomerulonephritis (C3GN) and CFHR5 nephropathy. These disorders share the key histological feature of isolated complement C3 deposits in the glomerulus. A common aetiology involving dysregulation of the alternative pathway (AP) of complement has been elucidated in the past decade, with genetic defects and/or autoantibodies able to be identified in a proportion of patients. We review the clinical and histological features of C3 glomerulopathy, relating these to underlying molecular mechanisms. The role of uncontrolled C3 activation in pathogenesis is emphasized, with important lessons from animal models. Methods, advantages and limitations of gene testing in the assessment of individuals or families with C3 glomerulopathy are discussed. While no therapy has yet been shown consistently effective, clinical evaluation of agents targeting specific components of the complement system is ongoing. However, limits to current knowledge regarding the natural history and the appropriate timing and duration of proposed therapies need to be addressed.