Primary Outcomes in Glomerulonephritis Study (PROGRESS)

肾小球肾炎研究的主要结果（进展）

• 批准号: 9115603
• 负责人: LAWRENCE B. HOLZMAN
• 金额: $ 98.02万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-16 至 2018-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9115603
• 关键词: Accounting; Adolescent; Adult; Affect; Age; Biological; Biological Markers; Biological Products; Biology; Biopsy; Caring; Categories; Child; Childhood; Chronic; Classification; Clinical; Clinical Data; Clinical Protocols; Clinical Research; Clinical Trials; Cohort Studies; Collaborations; Collection; Consensus; Consult; Data Collection; Diagnosis; Disease; Disease Progression; Early Diagnosis; Economic Burden; Eligibility Determination; Enrollment; Ensure; Epidemiology; Etiology; Focal Segmental Glomerulosclerosis; Foundations; Funding Agency; Genetic Markers; Genomics; Glomerulonephritis; Health Care Costs; Histopathology; Image; Immunoglobulin A; Incidence; Individual; Industry; International; Internet; Investigation; Kidney; Kidney Diseases; Link; Membranous Glomerulonephritis; Molecular; Morbidity - disease rate; Natural History; Nephrology; Observational Study; Outcome; Participant; Pathology; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Performance; Personnel Management; Phase; Pilot Projects; Population Heterogeneity; Prediction of Response to Therapy; Private Sector; Procedures; Proteinuria; Protocols documentation; Rare Diseases; Recording of previous events; Renal function; Renal glomerular disease; Research; Research Infrastructure; Research Personnel; Risk; Scientist; Specific qualifier value; System; Systems Biology; Target Populations; Therapeutic Intervention; Therapeutic Trials; Training and Infrastructure; base; biomarker discovery; biomarker identification; clinical biomarkers; clinical practice; clinical research site; cohort; data management; design; digital; disease classification; disease heterogeneity; disease natural history; experience; human biological material; improved; instrument; minimal risk; molecular marker; mortality; novel therapeutics; patient advocacy group; pediatric patients; primary outcome; prospective; public health relevance; response; social media; specific biomarkers; treatment response

DESCRIPTION (provided by applicant): Minimal Change Disease (MCD), Focal and Segmental Glomerulosclerosis (FSGS), Membranous Nephropathy (MN), and IgA Nephropathy (IgAN) are glomerular diseases that despite their rarity, account for a large fraction of prevalent ESKD. There is now consensus that the presently employed histopathologybased classification of these diseases is inadequate because it is not based on an understanding of the molecular basis of these diseases, and because it does not well predict the heterogeneous natural history or response to therapy of individuals within a given glomerular histopathological category. We propose that major barriers must be overcome before more effective interventional studies of primary chronic glomerular disease can be conducted or before patients suffering from these diseases can be properly diagnosed and treated. Among these barriers is the absence of well characterized specific biomarkers of glomerular disease that would allow refined, biologically relevant sub-classification of glomerular disease histopathology. Such disease sub-classification might overcome the effects of glomerular disease population heterogeneity that likely have complicated interpretation of past studies of these glomerular diseases. New glomerular disease biomarkers might also predict disease natural history, allow proper selection of and prediction of response to specific therapeutic intervention, allow early detection of disease, or provide indicators of disease activity. Importantly, a robust investigative infrastructure would facilitate collection, cultivation, and access to human biological material and associated clinical data necessary for biomarker identification, for developing instruments useful for evaluating patient reported outcomes (PRO) that might be useful as endpoints in therapeutic trials, and for conducting pilot clinical studies that would advance the care of MCD, FSGS, MN, IgAN patients. For these reasons, we propose the establishment of PROGRESS, a Participating Clinical Center (PCC) that brings together clinical and translational scientists, a lay research foundation, and partners from the private sector to study patients with MCD, FSGS, MN and IgAN as a collaborator in larger chronic glomerular disease research consortium.

描述（由申请人提供）：微小病变疾病（MCD）、局灶性和节段性肾小球硬化（FSGS）、膜性肾病（MN）和伊加肾病（IgAN）是肾小球疾病，尽管罕见，但占流行性肾小球疾病的很大一部分。 ESKD。目前的共识是，目前采用的基于组织病理学的这些疾病的分类是不够的，因为它不是基于对这些疾病的分子基础的理解，因为它不能很好地预测在给定的肾小球组织病理学类别内的个体的异质性自然史或对治疗的反应。我们建议，在对原发性慢性肾小球疾病进行更有效的干预性研究之前，或者在患有这些疾病的患者能够得到适当的诊断和治疗之前，必须克服主要障碍。在这些障碍中，缺乏肾小球疾病的良好表征的特异性生物标志物，其将允许肾小球疾病组织病理学的精细的、生物学相关的亚分类。这样的疾病亚分类可能克服肾小球疾病人群异质性的影响，这可能使这些肾小球疾病的过去研究的解释复杂化。新的肾小球疾病生物标志物还可以预测疾病的自然史，允许适当选择和预测对特定治疗干预的反应，允许早期检测疾病，或提供疾病活动的指标。重要的是，强有力的调查基础设施将有助于 收集、培养和获取生物标志物鉴定所需的人类生物材料和相关临床数据，用于开发可用于评价患者报告结局（PRO）的工具，这些结果可能用作治疗试验的终点，并用于进行试点临床研究，以促进MCD、FSGS、MN、IgAN患者的护理。出于这些原因，我们建议建立PROGRESS，一个参与临床中心（PCC），汇集临床和转化科学家，奠定研究基础， 与私营部门的合作伙伴一起研究MCD、FSGS、MN和IgAN患者，作为更大的慢性肾小球疾病研究联盟的合作者。