Vivarelli Marina, van de Kar Nicole, Labbadia Raffaella, Diomedi-Camassei Francesca, Thurman Joshua M
Division of Nephrology and Dialysis, Department of Pediatric Subspecialties, Bambino Gesù Pediatric Hospital IRCCS, Piazza S Onofrio 4, 00165, Rome, Italy.
Department of Pediatric Nephrology, Amalia Children's Hospital, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, The Netherlands.
Pediatr Nephrol. 2022 Mar;37(3):521-535. doi: 10.1007/s00467-021-05088-7. Epub 2021 May 18.
C3 glomerulopathy is a relatively new clinical entity that represents a challenge both to diagnose and to treat. As new therapeutic agents that act as complement inhibitors become available, many with an oral formulation, a better understanding of this disease and of the underlying complement dysregulation driving it has become increasingly useful to optimize patient care. Moreover, recent advances in research have clarified the role of complement in other glomerular diseases in which its role was less established, namely in immune-complex membranoproliferative glomerulonephritis (IC-MPGN), ANCA-vasculitis, IgA nephropathy, and idiopathic membranous nephropathy. Complement inhibitors are being studied in adult and adolescent clinical trials for these indications. This review summarizes current knowledge and future perspectives on every aspect of the diagnosis and management of C3 glomerulopathy and elucidates current understanding of the role of complement in this condition and in other glomerular diseases in children. An overview of ongoing trials involving therapeutic agents targeting complement in glomerular diseases is also provided.
C3肾小球病是一种相对较新的临床实体，在诊断和治疗方面都具有挑战性。随着作为补体抑制剂的新型治疗药物问世，其中许多为口服制剂，更深入地了解这种疾病以及驱动它的潜在补体失调对于优化患者护理变得越来越有用。此外，最近的研究进展阐明了补体在其他肾小球疾病中的作用，而在这些疾病中补体的作用此前尚不明确，即免疫复合物性膜增生性肾小球肾炎（IC-MPGN）、抗中性粒细胞胞浆抗体相关性血管炎、IgA肾病和特发性膜性肾病。目前正在针对这些适应症开展成人和青少年临床试验，研究补体抑制剂。本综述总结了C3肾小球病诊断和管理各方面的现有知识和未来展望，并阐明了目前对补体在这种疾病以及儿童其他肾小球疾病中作用的理解。还提供了涉及针对肾小球疾病补体的治疗药物的正在进行的试验概述。
