Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multidisciplinary approach

被引:252
作者
Gadegbeku, Crystal A. [1 ]
Gipson, Debbie S. [2 ,3 ,4 ,5 ]
Holzman, Lawrence B. [6 ,7 ]
Ojo, Akinlolu O. [2 ,3 ,4 ,5 ]
Song, Peter X. K. [2 ,3 ,4 ,5 ]
Barisoni, Laura [8 ,9 ,10 ]
Sampson, Matthew G. [2 ,3 ,4 ,5 ]
Kopp, Jeffrey B. [11 ]
Lemley, Kevin V. [12 ]
Nelson, Peter J. [13 ]
Lienczewski, Chrysta C. [2 ,3 ,4 ,5 ]
Adler, Sharon G. [14 ]
Appel, Gerald B. [16 ]
Cattran, Daniel C. [17 ]
Choi, Michael J. [18 ,19 ]
Contreras, Gabriel [8 ,9 ,10 ]
Dell, Katherine M. [20 ,21 ,22 ]
Fervenza, Fernando C. [23 ]
Gibson, Keisha L. [25 ,26 ]
Greenbaum, Larry A. [27 ,28 ]
Hernandez, Joel D. [29 ,30 ]
Hewitt, Stephen M. [31 ]
Hingorani, Sangeeta R. [32 ]
Hladunewich, Michelle [17 ]
Hogan, Marie C. [23 ]
Hogan, Susan L. [25 ,26 ]
Kaskel, Frederick J. [33 ]
Lieske, John C. [23 ,24 ]
Meyers, Kevin E. C. [6 ,7 ]
Nachman, Patrick H.
Nast, Cynthia C. [15 ]
Neu, Alicia M. [18 ,19 ]
Reich, Heather N. [17 ]
Sedor, John R. [20 ,21 ,22 ]
Sethna, Christine B. [34 ]
Trachtman, Howard [35 ,36 ]
Tuttle, Katherine R. [29 ,30 ]
Zhdanova, Olga [35 ,36 ]
Zilleruelo, Gaston E. [8 ,9 ,10 ]
Kretzler, Matthias [2 ,3 ,4 ,5 ]
机构
[1] Temple Univ, Dept Med, Philadelphia, PA 19122 USA
[2] Univ Michigan, Dept Nephrol Internal Med, Ann Arbor, MI 48109 USA
[3] Univ Michigan, Dept Computat Med & Bioinformat, Ann Arbor, MI 48109 USA
[4] Univ Michigan, Dept Pediat & Communicable Dis, Ann Arbor, MI 48109 USA
[5] Univ Michigan, Dept Biostat, Ann Arbor, MI 48109 USA
[6] Univ Penn, Dept Internal Med, Philadelphia, PA 19104 USA
[7] Univ Penn, Dept Pediat, Philadelphia, PA 19104 USA
[8] Univ Miami, Miller Sch Med, Dept Pathol, Miami, FL 33136 USA
[9] Univ Miami, Miller Sch Med, Dept Med, Miami, FL 33136 USA
[10] Univ Miami, Miller Sch Med, Dept Pediat, Miami, FL 33136 USA
[11] NIDDKD, Kidney Dis Sect, NIH, Bethesda, MD 20892 USA
[12] Univ So Calif, Dept Pediat, Div Nephrol, Childrens Hosp Los Angeles, Los Angeles, CA 90089 USA
[13] Univ Washington, Dept Med, Div Nephrol, Kidney Res Inst, Seattle, WA 98195 USA
[14] Univ Calif Los Angeles, Med Ctr, Dept Med, Los Angeles Biomed Res Inst Harbor, Los Angeles, CA 90024 USA
[15] Cedars Sinai Med Ctr, Dept Pathol, Los Angeles, CA 90048 USA
[16] Columbia Univ, Dept Med, Med Ctr, New York, NY USA
[17] Univ Toronto, Dept Med, Univ Hlth Network, Toronto, ON, Canada
[18] Johns Hopkins Univ, Dept Med, Baltimore, MD USA
[19] Johns Hopkins Univ, Dept Pediat, Baltimore, MD 21218 USA
[20] Case Western Reserve Univ, Dept Med, Cleveland, OH 44106 USA
[21] Case Western Reserve Univ, Dept Physiol & Biophys, Cleveland, OH 44106 USA
[22] Case Western Reserve Univ, Dept Pediat, Cleveland, OH 44106 USA
[23] Mayo Clin, Dept Internal Med, Div Nephrol & Hypertens, Rochester, MN USA
[24] Mayo Clin, Dept Lab Med & Pathol, Rochester, MN USA
[25] Univ N Carolina, Dept Med, Chapel Hill, NC USA
[26] Univ N Carolina, Dept Pediat, Chapel Hill, NC USA
[27] Emory Univ, Dept Pediat, Atlanta, GA 30322 USA
[28] Childrens Healthcare Atlanta, Atlanta, GA USA
[29] Sacred Heart Med Ctr, Dept Med, Providence Med Res Ctr, Spokane, WA USA
[30] Sacred Heart Med Ctr, Dept Pediat, Providence Med Res Ctr, Spokane, WA USA
[31] NCI, Pathol Lab, NIH, Bethesda, MD 20892 USA
[32] Univ Washington, Dept Pediat, Div Nephrol, Seattle Childrens Hosp, Seattle, WA 98195 USA
[33] Montefiore Med Ctr, Dept Pediat, Bronx, NY 10467 USA
[34] Cohen Childrens Med Ctr New York, Dept Pediat, New Hyde Pk, NY USA
[35] NYU, Dept Pediat, Sch Med, New York, NY 10016 USA
[36] NYU, Sch Med, Dept Med, New York, NY 10016 USA
关键词
clinical trial; focal segmental glomerulosclerosis; membranous nephropathy; nephrotic syndrome; FOCAL SEGMENTAL GLOMERULOSCLEROSIS; IDIOPATHIC MEMBRANOUS NEPHROPATHY; GENETIC-HETEROGENEITY; CLINICAL SPECTRUM; RENAL-DISEASE; CLASSIFICATION; BIOLOGY;
D O I
10.1038/ki.2012.428
中图分类号
R5 [内科学]; R69 [泌尿科学(泌尿生殖系疾病)];
学科分类号
1002 ; 100201 ;
摘要
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. Kidney International (2013) 83, 749-756; doi:10.1038/ki.2012.428; published online 16 January 2013
引用
收藏
页码:749 / 756
页数:8
相关论文
共 40 条
[1]   Current views on collapsing glomerulopathy [J].
Albaqumi, Mamdouh ;
Barisoni, Laura .
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2008, 19 (07) :1276-1281
[2]  
[Anonymous], 2002, Statistical Methods in Diagnostic Medicine
[3]   A proposed taxonomy for the podocytopathies: A reassessment of the primary nephrotic diseases [J].
Barisoni, Laura ;
Schnaper, H. William ;
Kopp, Jeffrey B. .
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2007, 2 (03) :529-542
[4]   Novel Quantitative Method to Evaluate Globotriaosylceramide Inclusions in Renal Peritubular Capillaries by Virtual Microscopy in Patients With Fabry Disease [J].
Barisoni, Laura ;
Jennette, J. Charles ;
Colvin, Robert ;
Sitaraman, Sheela ;
Bragat, Alexander ;
Castelli, Jeff ;
Walker, Dan ;
Boudes, Pol .
ARCHIVES OF PATHOLOGY & LABORATORY MEDICINE, 2012, 136 (07) :816-824
[5]  
CAMERON JS, 1992, NEPHROL DIAL TRANSPL, V7, P72
[6]   The Oxford classification of IgA nephropathy: rationale, clinicopathological correlations, and classification [J].
Cattran, Daniel C. ;
Coppo, Rosanna ;
Cook, H. Terence ;
Feehally, John ;
Roberts, Ian S. D. ;
Troyanov, Stephan ;
Alpers, Charles E. ;
Amore, Alessandro ;
Barratt, Jonathan ;
Berthoux, Francois ;
Bonsib, Stephen ;
Bruijn, Jan A. ;
D'Agati, Vivette ;
D'Amico, Giuseppe ;
Emancipator, Steven ;
Emma, Francesco ;
Ferrario, Franco ;
Fervenza, Fernando C. ;
Florquin, Sandrine ;
Fogo, Agnes ;
Geddes, Colin C. ;
Groene, Hermann-Josef ;
Haas, Mark ;
Herzenberg, Andrew M. ;
Hill, Prue A. ;
Hogg, Ronald J. ;
Hsu, Stephen I. ;
Jennette, J. Charles ;
Joh, Kensuke ;
Julian, Bruce A. ;
Kawamura, Tetsuya ;
Lai, Fernand M. ;
Leung, Chi Bon ;
Li, Lei-Shi ;
Li, Philip K. T. ;
Liu, Zhi-Hong ;
Mackinnon, Bruce ;
Mezzano, Sergio ;
Schena, F. Paolo ;
Tomino, Yasuhiko ;
Walker, Patrick D. ;
Wang, Haiyan ;
Weening, Jan J. ;
Yoshikawa, Nori ;
Zhang, Hong .
KIDNEY INTERNATIONAL, 2009, 76 (05) :534-545
[7]   Validation of a predictive model of idiopathic membranous nephropathy: Its clinical and research implications [J].
Cattran, DC ;
Pei, Y ;
Greenwood, CMT ;
Ponticelli, C ;
Passerini, P ;
Honkanen, E .
KIDNEY INTERNATIONAL, 1997, 51 (03) :901-907
[8]  
Diggle P., 2002, ANAL LONGITUDINAL DA
[9]   COQ2 nephropathy:: A newly described inherited mitochondriopathy with primary renal involvement [J].
Diomedi-Camassei, Francesca ;
Di Giandomenico, Silvia ;
Santorelli, Filippo M. ;
Caridi, Gianluca ;
Piemonte, Fiorella ;
Montini, Giovanni ;
Ghiggeri, Gian Marco ;
Murer, Luisa ;
Barisoni, Laura ;
Pastore, Anna ;
Muda, Andrea Onetti ;
Valente, Maria Luisa ;
Bertini, Enrico ;
Emma, Francesco .
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2007, 18 (10) :2773-2780
[10]   Sample size planning for developing classifiers using high-dimensional DNA microarray data [J].
Dobbin, Kevin K. ;
Simon, Richard M. .
BIOSTATISTICS, 2007, 8 (01) :101-117