Division of Nephrology, Department of Medicine, Columbia University, New York, NY.
Division of Nephrology, Department of Medicine, University of Iowa, Iowa City, IA.
Adv Kidney Dis Health. 2024 May;31(3):223-233. doi: 10.1053/j.akdh.2024.05.002.
C3 glomerulopathy (C3G) is a rare disorder marked by deposition of C3 in the glomerulus, resulting in damage to the glomerular filtration unit and presenting with features of the nephritic and nephrotic syndromes. Fundamentally, C3G is caused by dysregulation of the alternative pathway of the complement cascade, either due to genetic variants or acquired humoral factors. Despite significant advances in recent years in the understanding of the underlying mechanisms and culprit lesions that result in the development of C3G, treatment options remain severely limited, and the prognosis is often poor. Fortunately, a number of anticomplement therapies are emerging from the drug development pipeline, with several in late-stage testing in patients with C3G, and there is hope that we will soon have more targeted options for managing patients with this devastating disease. In this review, we provide an overview of C3G, as well as summarizing the evidence for current treatments and detailing the clinical trials that are currently underway.
C3 肾小球病（C3G）是一种罕见的疾病，其特征是肾小球中 C3 的沉积，导致肾小球滤过单位的损伤，并表现出肾炎和肾病综合征的特征。从根本上说，C3G 是由于补体级联的替代途径的调节紊乱引起的，要么是由于遗传变异，要么是由于获得性体液因素。尽管近年来在理解导致 C3G 发展的潜在机制和罪魁祸首病变方面取得了重大进展，但治疗选择仍然非常有限，预后往往较差。幸运的是，许多抗补体疗法正在从药物开发渠道中涌现，有几种在 C3G 患者的后期测试中，人们希望我们很快就能为管理这种破坏性疾病的患者提供更有针对性的选择。在这篇综述中，我们提供了 C3G 的概述，总结了目前的治疗方法的证据，并详细介绍了目前正在进行的临床试验。
