TY  - JOUR
ID  - discovery10081736
VL  - 4
JF  - Kidney International Reports
N1  - This is an open access article under the CC BYNC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
N2  - Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic
cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the past
decade, it has become more widely appreciated that the disease course begins in childhood. However,
evidence-based guidelines on how to manage and approach children diagnosed with or at risk of ADPKD
are lacking. Also, scoring systems to stratify patients into risk categories have been established only for
adults. Overall, there are insufficient data on the clinical course during childhood. We therefore initiated
the global ADPedKD project to establish a large international pediatric ADPKD cohort for deep
characterization.
Methods: Global ADPedKD is an international multicenter observational study focusing on childhooddiagnosed ADPKD. This collaborative project is based on interoperable Web-based databases,
comprising 7 regional and independent but uniformly organized chapters, namely Africa, Asia, Australia,
Europe, North America, South America, and the United Kingdom. In the database, a detailed basic data
questionnaire, including genetics, is used in combination with data entry from follow-up visits, to provide
both retrospective and prospective longitudinal data on clinical, radiologic, and laboratory findings, as well
as therapeutic interventions.
Discussion: The global ADPedKD initiative aims to characterize in detail the most extensive international
pediatric ADPKD cohort reported to date, providing evidence for the development of unified diagnostic,
follow-up, and treatment recommendations regarding modifiable disease factors. Moreover, this registry
will serve as a platform for the development of clinical and/or biochemical markers predicting the risk of
early and progressive disease.
Y1  - 2019/09//
EP  - 1284
SN  - 2468-0249
KW  - ADPKD; ADPedKD Registry; children; longitudinal
IS  - 9
A1  - De Rechter, S
A1  - Bockenhauer, D
A1  - Guay-Woodford, LM
A1  - Liu, I
A1  - Mallett, AJ
A1  - Soliman, NA
A1  - Sylvestre, LC
A1  - Schaefer, F
A1  - Liebau, MC
A1  - Mekahli, D
A1  - Adamczyk, P
A1  - Akinci, N
A1  - Alpay, H
A1  - Ardelean, C
A1  - Ayasreh, N
A1  - Aydin, Z
A1  - Bael, A
A1  - Baudouin, V
A1  - Bayrakci, US
A1  - Bensman, A
A1  - Bialkevich, H
A1  - Biebuyck, A
A1  - Boyer, O
A1  - Bjanid, O
A1  - Bry?ka, A
A1  - Çal??kan, S
A1  - Cambier, A
A1  - Camelio, A
A1  - Carbone, V
A1  - Charbit, M
A1  - Chiodini, B
A1  - Chirita, A
A1  - Çiçek, N
A1  - Cerkauskiene, R
A1  - Collard, L
A1  - Conceiçao, M
A1  - Constantinescu, I
A1  - Couderc, A
A1  - Crapella, B
A1  - Cvetkovic, M
A1  - Dima, B
A1  - Diomeda, F
A1  - Docx, M
A1  - Dolan, N
A1  - Dossier, C
A1  - Drozdz, D
A1  - Drube, J
A1  - Dunand, O
A1  - Dusan, P
A1  - Eid, LA
A1  - Emma, F
A1  - Espino Hernandez, M
A1  - Fila, M
A1  - Furlano, M
A1  - Gafencu, M
A1  - Ghuysen, MS
A1  - Giani, M
A1  - Giordano, M
A1  - Girisgen, I
A1  - Godefroid, N
A1  - Godron-Dubrasquet, A
A1  - Gojkovic, I
A1  - Gonzalez, E
A1  - Gökçe, I
A1  - Groothoff, JW
A1  - Guarino, S
A1  - Guffens, A
A1  - Hansen, P
A1  - Harambat, J
A1  - Haumann, S
A1  - He, G
A1  - Heidet, L
A1  - Helmy, R
A1  - Hemery, F
A1  - Hooman, N
A1  - llanas, B
A1  - Jankauskiene, A
A1  - Janssens, P
A1  - Karamaria, S
A1  - Kazyra, I
A1  - Koenig, J
A1  - Krid, S
A1  - Krug, P
A1  - Kwon, V
A1  - La Manna, A
A1  - Leroy, V
A1  - Litwin, M
A1  - Lombet, J
A1  - Longo, G
A1  - Lungu, AC
A1  - Mallawaarachchi, A
A1  - Marin, A
A1  - Marzuillo, P
A1  - Massella, L
A1  - Mastrangelo, A
A1  - McCarthy, H
A1  - Miklaszewska, M
A1  - Moczulska, A
A1  - Montini, G
A1  - Morawiec-Knysak, A
SP  - 1271
AV  - public
TI  - ADPedKD: A Global Online Platform on the Management of Children With ADPKD
UR  - https://doi.org/10.1016/j.ekir.2019.05.015
ER  -