TY - JOUR
T1 - Pediatric contributions and lessons learned from the NEPTUNE cohort study
AU - for the NEPTUNE investigators
AU - Modi, Zubin J.
AU - Zhai, Yan
AU - Yee, Jennifer
AU - Desmond, Hailey
AU - Hao, Wei
AU - Sampson, Matthew G.
AU - Sethna, Christine B.
AU - Wang, Chia Shi
AU - Gipson, Debbie S.
AU - Trachtman, Howard
AU - Kretzler, Matthias
AU - Nast, Cynthia
AU - Zuo, Yiqin
AU - Yin, Hong
AU - Yamashita, Michifumi
AU - Wu, Ming
AU - Thomas, David
AU - Stokes, Barry
AU - Sekulik, Miroslav
AU - Royal, Virginie
AU - Rosenberg, Avi
AU - Palmer, Matthew
AU - Najafian, Behzad
AU - Moreno, Vanessa
AU - Mikhailov, Alexei
AU - Messias, Nidia
AU - Lemley, Kevin
AU - Kambham, Neeraja
AU - Holanda, Danni
AU - Hodgin, Jeff
AU - Hewitt, Stephen
AU - Herlitz, Leal
AU - Hassler, Jared
AU - Demeke, Dawit
AU - Cassol, Clarissa
AU - Caltharp, Shelley
AU - Bu, Lihong
AU - Bagnasco, Serena
AU - Avila-Casado, Carmen
AU - Zee, Jarcy
AU - Smith, Abigail
AU - Barisoni, Laura
AU - Gadegbeku, Crystal
AU - Li, Shannon
AU - Li, Shengqian
AU - Kincaid, Colleen
AU - Williams, Amanda
AU - Troost, Jonathan
AU - Scherr, Rebecca
AU - Lienczewski, Chrysta
AU - Larkina, Maria
AU - Ju, Wenjun
AU - Fermin, Damian
AU - Eddy, Sean
AU - Arbit, Michael
AU - Alter, Gabrielle
AU - Mainieri, Tina
AU - Salmon, Eloise
AU - Gillespie, Brenda
AU - Dharnidharka, Vikas
AU - Lin, Jen Jar
AU - Kuykendall, Kelli
AU - Pao, Emily
AU - Manahan, Linda
AU - Tuttle, Katherine
AU - Hingorani, Sangeeta
AU - Jefferson, Ashley
AU - Hellewege, Jamie
AU - Brown, Elizabeth
AU - Sambandam, Kamalanathan
AU - Roehm, Elizabeth
AU - Sharma, Samin
AU - Edwards, Aliya
AU - Kallem, Krishna
AU - Meyers, Kevin
AU - Holzman, Lawrence
AU - Kelley, Sara
AU - Froment, Anne
AU - Gibson, Keisha
AU - Derebail, Vimal
AU - Rauwolf, Nicolas
AU - Kowalski, Amy
AU - Rheault, Michelle
AU - Nachman, Patrick
AU - Ni, Roxy
AU - Williams, A.
AU - Mariani, Laura
AU - Bandes, Miguel
AU - Fornoni, Alessia
AU - Creed, Catherine
AU - McCarthy, Ellen
AU - Rogers, Nathan
AU - Dylewski, James
AU - Brakeman, Paul
AU - Romano, Martin
AU - Ling, Paul
AU - Hladunewich, Michelle
AU - Reich, Heather
AU - Batla, Sadaf
AU - Shah, Shweta
AU - Lee, Iris
AU - Dave, Shiktij
AU - Lafayette, Richard
AU - Roberts, Laci
AU - Almaani, Salem
AU - Miranda, Melanie
AU - Pehrson, Laura Jane
AU - Zhdanova, Olga
AU - Malaga-Dieguez, Laura
AU - Flynn, Patricia
AU - Kaskel, Frederick
AU - Ross, Michael
AU - Reidy, Kim
AU - Alston, Cheryl
AU - Selewski, David
AU - Fervenza, Fernando
AU - Hogan, Marie
AU - Lieske, John
AU - Williams, Miahje
AU - Atkinson, Meredith
AU - Itteera, Mathew
AU - Amarah, Amatur
AU - LaPage, Janine
AU - Adler, Sharon
AU - Yun, Emily
AU - Greenbaum, Laurence
AU - Smith, Maurice
AU - Gbadegesin, Rasheed
AU - Olabisi, Opeyemi
AU - Canetta, Pietro
AU - Vento, Suzanne
AU - Markus, Kelsey
AU - Srivastava, Tarak
AU - Sharma, Silpa
AU - Macumber, Ian
AU - Martin, Blair
AU - Sedor, John
AU - O’Toole, John
AU - Dell, Katherine
AU - Lo, Layla
AU - Massengill, Susan
N1 - Publisher Copyright:
© The Author(s), under exclusive licence to International Pediatric Nephrology Association 2024.
PY - 2024/9
Y1 - 2024/9
N2 - Primary glomerular diseases are rare entities. This has hampered efforts to better understand the underlying pathobiology and to develop novel safe and effective therapies. NEPTUNE is a rare disease network that is focused on patients of all ages with minimal change disease, focal segmental glomerulosclerosis, and membranous nephropathy. It is a longitudinal cohort study that collects detailed demographic, clinical, histopathologic, genomic, transcriptomic, and metabolomic data. The goal is to develop a molecular classification for these disorders that supersedes the traditional pathological features-based schema. Pediatric patients are important contributors to this ongoing project. In this review, we provide a snapshot of the children and adolescents enrolled in NEPTUNE and summarize some key observations that have been made based on the data accumulated during the study. In addition, we describe the development of NEPTUNE Match, a program that aims to leverage the multi-scalar information gathered for each individual patient to provide guidance about potential clinical trial participation based on the molecular characterization and non-invasive biomarker profile. This represents the first organized effort to apply principles of precision medicine to the treatment of patients with primary glomerular disease. NEPTUNE has proven to be an invaluable asset in the study of glomerular diseases in patients of all ages including children and adolescents.
AB - Primary glomerular diseases are rare entities. This has hampered efforts to better understand the underlying pathobiology and to develop novel safe and effective therapies. NEPTUNE is a rare disease network that is focused on patients of all ages with minimal change disease, focal segmental glomerulosclerosis, and membranous nephropathy. It is a longitudinal cohort study that collects detailed demographic, clinical, histopathologic, genomic, transcriptomic, and metabolomic data. The goal is to develop a molecular classification for these disorders that supersedes the traditional pathological features-based schema. Pediatric patients are important contributors to this ongoing project. In this review, we provide a snapshot of the children and adolescents enrolled in NEPTUNE and summarize some key observations that have been made based on the data accumulated during the study. In addition, we describe the development of NEPTUNE Match, a program that aims to leverage the multi-scalar information gathered for each individual patient to provide guidance about potential clinical trial participation based on the molecular characterization and non-invasive biomarker profile. This represents the first organized effort to apply principles of precision medicine to the treatment of patients with primary glomerular disease. NEPTUNE has proven to be an invaluable asset in the study of glomerular diseases in patients of all ages including children and adolescents.
KW - Biomarker
KW - Glomerular disease
KW - Kidney biopsy
KW - Nephrotic syndrome
KW - Pediatrics
KW - Precision medicine
UR - http://www.scopus.com/inward/record.url?scp=85182435061&partnerID=8YFLogxK
U2 - 10.1007/s00467-023-06256-7
DO - 10.1007/s00467-023-06256-7
M3 - Review article
C2 - 38233720
AN - SCOPUS:85182435061
SN - 0931-041X
VL - 39
SP - 2555
EP - 2568
JO - Pediatric Nephrology
JF - Pediatric Nephrology
IS - 9
ER -