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确立C3肾小球病临床结局的未来方向:来自患者和医生的观点

Establishing the Future Direction of Clinical Outcomes in C3 Glomerulopathy: Perspectives From a Patient and a Physician.

作者信息

Java Anuja, Fuller Lindsey

机构信息

Department of Nephrology, Washington University School of Medicine in St. Louis, St. Louis, MO.

Blue Mound, IL.

出版信息

Kidney Med. 2024 Nov 13;7(1):100928. doi: 10.1016/j.xkme.2024.100928. eCollection 2025 Jan.

Abstract

Complement 3 glomerulopathy (C3G) is an ultra-rare glomerulonephritis caused by dysregulation of the alternative complement pathway. C3G has an estimated incidence of 1-3 cases per million people in the United States. Diagnosing C3G based solely on clinical and laboratory features is challenging because it mimics several other glomerular diseases; therefore, diagnosis requires a kidney biopsy. In the absence of disease-modifying therapies and optimal patient management strategies, C3G poses a significant physical and emotional burden on patients and caregivers. Common symptoms of glomerulonephritis include fatigue, edema, anxiety, and/or depression, which have profound effects on patients' daily lives. Approximately half of all patients progress to kidney failure within 10 years of diagnosis. Encouragingly, the treatment landscape in C3G is poised to change, with several targeted complement inhibitors in late-stage development. This perspectives article explores a patient's journey in C3G and discusses the current and future status of clinical outcomes and patient management from the viewpoints of a practicing nephrologist and a patient.

摘要

补体3肾小球病(C3G)是一种由替代补体途径失调引起的极为罕见的肾小球肾炎。在美国,C3G的估计发病率为每百万人中有1至3例。仅根据临床和实验室特征诊断C3G具有挑战性,因为它与其他几种肾小球疾病相似;因此,诊断需要进行肾活检。在缺乏疾病改善疗法和最佳患者管理策略的情况下,C3G给患者和护理人员带来了巨大的身体和情感负担。肾小球肾炎的常见症状包括疲劳、水肿、焦虑和/或抑郁,这些对患者的日常生活有深远影响。大约一半的患者在诊断后10年内会发展为肾衰竭。令人鼓舞的是,C3G的治疗格局即将改变,有几种靶向补体抑制剂正处于后期开发阶段。这篇观点文章探讨了C3G患者的历程,并从执业肾病学家和患者的角度讨论了临床结局和患者管理的现状与未来。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ff2c/11699595/0601df12debb/gr1.jpg

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