Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multidisciplinary approach.
СтатьяМногоцентровое исследованиеПоддержка NIH: внешняя программаПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод в очереди
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Gadegbeku Crystal A, Gipson Debbie S, Holzman Lawrence B, Ojo Akinlolu O, Song Peter X K, Barisoni Laura, Sampson Matthew G, Kopp Jeffrey B, Lemley Kevin V, Nelson Peter J, Lienczewski Chrysta C, Adler Sharon G, Appel Gerald B, Cattran Daniel C, Choi Michael J, Contreras Gabriel, Dell Katherine M, Fervenza Fernando C, Gibson Keisha L, Greenbaum Larry A, Hernandez Joel D, Hewitt Stephen M, Hingorani Sangeeta R, Hladunewich Michelle, Hogan Marie C, Hogan Susan L, Kaskel Frederick J, Lieske John C, Meyers Kevin E C, Nachman Patrick H, Nast Cynthia C, Neu Alicia M, Reich Heather N, Sedor John R, Sethna Christine B, Trachtman Howard, Tuttle Katherine R, Zhdanova Olga, Zilleruelo Gastòn E, Kretzler Matthias
Аннотация
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.