肾病综合征研究网络的临床结局：疾病负担及随时间变化的治疗模式
Clinical Outcomes in the Nephrotic Syndrome Study Network: Disease Burden and Treatment Patterns over Time.
作者信息
Drexler Yelena, Athavale Ambarish, Smith Abigail R, Liu Qian, Zee Jarcy, Mariani Laura H, Lafayette Richard A
机构信息
Katz Family Division of Nephrology and Hypertension, Department of Medicine, University of Miami Miller School of Medicine, Miami, FL, USA.
Division of Nephrology-Hypertension, Department of Medicine, University of California San Diego, La Jolla, CA, USA.
出版信息
Glomerular Dis. 2025 Jun 5;5(1):288-303. doi: 10.1159/000546655. eCollection 2025 Jan-Dec.
INTRODUCTION
Among patients with proteinuric glomerular diseases, there is a paucity of high-quality evidence and substantial variation in practice patterns among nephrologists. Our objective was to describe the clinical presentation, treatment patterns, and outcomes of patients with biopsy-proven glomerular diseases in a contemporary, well-phenotyped, diverse cohort.
METHODS
The Nephrotic Syndrome Study Network (NEPTUNE) is a prospective observational cohort study of children and adults with proteinuric glomerular diseases enrolled at 23 centers in the USA and Canada since 2009. We included participants who underwent their first clinically indicated kidney biopsy demonstrating minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), or membranous nephropathy (MN). We described demographic and clinical characteristics at the time of biopsy and baseline visits. We analyzed treatment patterns for participants with and without immunosuppressive therapy (IST) use prior to biopsy. We described clinical outcomes including complete remission (CR) and proteinuria, stratified by IST use, at biopsy and up to 36 months' follow-up.
RESULTS
Among 507 NEPTUNE participants who underwent biopsy, 203 were classified as having FSGS, 193 as having MCD, and 111 as having MN. Corticosteroid exposure was high overall and highest among MCD patients. Substantial heterogeneity in treatment choices was evident, particularly among those initiating second-line therapy. The rate of kidney failure was highest, and CR rates were lowest, among patients with FSGS, who experienced ∼50% cumulative probability of first remission at 36 months after biopsy. At 36 months, 49.5% of all patients were not in CR; 19.3% were not in CR despite being on IST. Additionally, 20.2% of patients had proteinuria >1.5 g/g; among those on IST at their 36-month visit, 26.3% had UPCR >1.5 g/g.
CONCLUSION
A substantial proportion of patients were not in remission and had persistent proteinuria despite being on IST 3 years after their first biopsy.
引言
在患有蛋白尿性肾小球疾病的患者中，高质量证据匮乏，肾脏科医生的实践模式存在很大差异。我们的目标是在一个当代的、特征明确的、多样化队列中描述经活检证实的肾小球疾病患者的临床表现、治疗模式和结局。
方法
肾病综合征研究网络（NEPTUNE）是一项前瞻性观察性队列研究，自2009年起在美国和加拿大的23个中心招募患有蛋白尿性肾小球疾病的儿童和成人。我们纳入了首次接受临床指征肾脏活检显示为微小病变病（MCD）、局灶节段性肾小球硬化（FSGS）或膜性肾病（MN）的参与者。我们描述了活检时和基线访视时的人口统计学和临床特征。我们分析了活检前使用和未使用免疫抑制治疗（IST）的参与者的治疗模式。我们描述了临床结局，包括完全缓解（CR）和蛋白尿情况，按IST使用情况分层，在活检时以及长达36个月的随访中。
结果
在507名接受活检的NEPTUNE参与者中，203人被分类为患有FSGS，193人患有MCD，111人患有MN。总体而言，皮质类固醇暴露率较高，在MCD患者中最高。治疗选择存在显著异质性，尤其是在开始二线治疗的患者中。FSGS患者的肾衰竭发生率最高，CR率最低，他们在活检后36个月时首次缓解的累积概率约为50%。在36个月时，所有患者中有49.5%未达到CR；19.3%尽管接受了IST治疗仍未达到CR。此外，20.2%的患者蛋白尿>1.5 g/g；在36个月访视时接受IST治疗的患者中，26.3%的尿蛋白肌酐比值（UPCR）>1.5 g/g。
结论
很大一部分患者在首次活检3年后尽管接受了IST治疗仍未缓解且存在持续性蛋白尿。
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