Department of Medicine, Temple University, Philadelphia, Pennsylvania, USA.
Kidney Int. 2013 Apr;83(4):749-56. doi: 10.1038/ki.2012.428. Epub 2013 Jan 16.
The Nephrotic Syndrome Study Network (NEPTUNE) is a North American multicenter collaborative consortium established to develop a translational research infrastructure for nephrotic syndrome. This includes a longitudinal observational cohort study, a pilot and ancillary study program, a training program, and a patient contact registry. NEPTUNE will enroll 450 adults and children with minimal change disease, focal segmental glomerulosclerosis, and membranous nephropathy for detailed clinical, histopathological, and molecular phenotyping at the time of clinically indicated renal biopsy. Initial visits will include an extensive clinical history, physical examination, collection of urine, blood and renal tissue samples, and assessments of quality of life and patient-reported outcomes. Follow-up history, physical measures, urine and blood samples, and questionnaires will be obtained every 4 months in the first year and biannually, thereafter. Molecular profiles and gene expression data will be linked to phenotypic, genetic, and digitalized histological data for comprehensive analyses using systems biology approaches. Analytical strategies were designed to transform descriptive information to mechanistic disease classification for nephrotic syndrome and to identify clinical, histological, and genomic disease predictors. Thus, understanding the complexity of the disease pathogenesis will guide further investigation for targeted therapeutic strategies.
肾病综合征研究网络(NEPTUNE)是一个北美的多中心合作联盟,旨在为肾病综合征建立转化研究基础设施。这包括一个纵向观察队列研究、一个试点和辅助研究计划、一个培训计划和一个患者联系登记处。NEPTUNE 将招募 450 名患有微小病变性肾病、局灶节段性肾小球硬化症和膜性肾病的成年人和儿童,在临床指征明确的肾活检时进行详细的临床、组织病理学和分子表型分析。初次就诊将包括详细的临床病史、体格检查、尿液、血液和肾组织样本采集,以及生活质量和患者报告结果的评估。在第一年的每 4 个月和之后的每 6 个月将获得随访的病史、身体测量、尿液和血液样本以及问卷调查。分子谱和基因表达数据将与表型、遗传和数字化组织学数据相关联,以使用系统生物学方法进行综合分析。分析策略旨在将描述性信息转化为肾病综合征的机制疾病分类,并确定临床、组织学和基因组疾病预测因子。因此,了解疾病发病机制的复杂性将指导针对治疗策略的进一步研究。