Kidney International Reports (Sep 2019)
ADPedKD: A Global Online Platform on the Management of Children With ADPKD
- Stéphanie De Rechter,
- Detlef Bockenhauer,
- Lisa M. Guay-Woodford,
- Isaac Liu,
- Andrew J. Mallett,
- Neveen A. Soliman,
- Lucimary C. Sylvestre,
- Franz Schaefer,
- Max C. Liebau,
- Djalila Mekahli,
- P. Adamczyk,
- N. Akinci,
- H. Alpay,
- C. Ardelean,
- N. Ayasreh,
- Z. Aydin,
- A. Bael,
- V. Baudouin,
- U.S. Bayrakci,
- A. Bensman,
- H. Bialkevich,
- A. Biebuyck,
- O. Boyer,
- O. Bjanid,
- O. Boyer,
- A. Bryłka,
- S. Çalışkan,
- A. Cambier,
- A. Camelio,
- V. Carbone,
- M. Charbit,
- B. Chiodini,
- A. Chirita,
- N. Çiçek,
- R. Cerkauskiene,
- L. Collard,
- M. Conceiçao,
- I. Constantinescu,
- A. Couderc,
- B. Crapella,
- M. Cvetkovic,
- B. Dima,
- F. Diomeda,
- M. Docx,
- N. Dolan,
- C. Dossier,
- D. Drozdz,
- J. Drube,
- O. Dunand,
- P. Dusan,
- L.A. Eid,
- F. Emma,
- M. Espino Hernandez,
- M. Fila,
- M. Furlano,
- M. Gafencu,
- M.S. Ghuysen,
- M. Giani,
- M. Giordano,
- I. Girisgen,
- N. Godefroid,
- A. Godron-Dubrasquet,
- I. Gojkovic,
- E. Gonzalez,
- I. Gökçe,
- J.W. Groothoff,
- S. Guarino,
- A. Guffens,
- P. Hansen,
- J. Harambat,
- S. Haumann,
- G. He,
- L. Heidet,
- R. Helmy,
- F. Hemery,
- N. Hooman,
- B. llanas,
- A. Jankauskiene,
- P. Janssens,
- S. Karamaria,
- I. Kazyra,
- J. Koenig,
- S. Krid,
- P. Krug,
- V. Kwon,
- A. La Manna,
- V. Leroy,
- M. Litwin,
- J. Lombet,
- G. Longo,
- A.C. Lungu,
- A. Mallawaarachchi,
- A. Marin,
- P. Marzuillo,
- L. Massella,
- A. Mastrangelo,
- H. McCarthy,
- M. Miklaszewska,
- A. Moczulska,
- G. Montini,
- A. Morawiec-Knysak,
- D. Morin,
- L. Murer,
- I. Negru,
- F. Nobili,
- L. Obrycki,
- H. Otoukesh,
- S. Özcan,
- L. Pape,
- S. Papizh,
- P. Parvex,
- M. Pawlak-Bratkowska,
- L. Prikhodina,
- A. Prytula,
- C. Quinlan,
- A. Raes,
- B. Ranchin,
- N. Ranguelov,
- R. Repeckiene,
- C. Ronit,
- R. Salomon,
- R. Santagelo,
- S.K. Saygılı,
- S. Schaefer,
- M. Schreuder,
- T. Schurmans,
- T. Seeman,
- N. Segers,
- M. Sinha,
- E. Snauwaert,
- B. Spasojevic,
- S. Stabouli,
- C. Stoica,
- R. Stroescu,
- E. Szczepanik,
- M. Szczepańska,
- K. Taranta-Janusz,
- A. Teixeira,
- J. Thumfart,
- M. Tkaczyk,
- R. Torra,
- D. Torres,
- N. Tram,
- B. Utsch,
- J. Vande Walle,
- R. Vieux,
- R. Vitkevic,
- A. Wilhelm-Bals,
- E. Wühl,
- Z.Y. Yildirim,
- S. Yüksel,
- K. Zachwieja
Affiliations
- Stéphanie De Rechter
- Department of Pediatric Nephrology, University Hospitals Leuven, Leuven, Belgium; PKD Research Group, Department of Development and Regeneration, KU Leuven, Leuven, Leuven, Belgium
- Detlef Bockenhauer
- UCL Centre for Nephrology, London, United Kingdom; Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
- Lisa M. Guay-Woodford
- Center for Translational Science, Children's National Health System, Washington, District of Columbia, USA
- Isaac Liu
- Khoo Teck Puat–National University Children's Medical Institute, National University Health System, Singapore, Singapore
- Andrew J. Mallett
- Kidney Health Service and Conjoint Renal Research Laboratory, Royal Brisbane and Women's Hospital, Brisbane, Australia; Faculty of Medicine and Institute for Molecular Bioscience, The University of Queensland, Brisbane, Australia; The KidGen Collaborative and Australian Genomics Health Alliance, Melbourne, Australia
- Neveen A. Soliman
- Department of Pediatrics, Centre of Pediatric Nephrology and Transplantation, Kasr Al Ainy School of Medicine, Cairo University, Cairo, Egypt
- Lucimary C. Sylvestre
- Hospital Pequeno Príncipe, Curitiba, Brazil
- Franz Schaefer
- Division of Pediatric Nephrology, Centre for Pediatrics and Adolescent Medicine, Heidelberg University Medical Centre, Heidelberg, Germany
- Max C. Liebau
- Department of Pediatrics and Center for Molecular Medicine, University Hospital of Cologne, Cologne, Germany
- Djalila Mekahli
- Department of Pediatric Nephrology, University Hospitals Leuven, Leuven, Belgium; PKD Research Group, Department of Development and Regeneration, KU Leuven, Leuven, Leuven, Belgium; Correspondence: Djalila Mekahli, Department of Pediatric Nephrology, University Hospitals Leuven, Herestraat 49, 3000 Leuven, Belgium.
- P. Adamczyk
- N. Akinci
- H. Alpay
- C. Ardelean
- N. Ayasreh
- Z. Aydin
- A. Bael
- V. Baudouin
- U.S. Bayrakci
- A. Bensman
- H. Bialkevich
- A. Biebuyck
- O. Boyer
- O. Bjanid
- O. Boyer
- A. Bryłka
- S. Çalışkan
- A. Cambier
- A. Camelio
- V. Carbone
- M. Charbit
- B. Chiodini
- A. Chirita
- N. Çiçek
- R. Cerkauskiene
- L. Collard
- M. Conceiçao
- I. Constantinescu
- A. Couderc
- B. Crapella
- M. Cvetkovic
- B. Dima
- F. Diomeda
- M. Docx
- N. Dolan
- C. Dossier
- D. Drozdz
- J. Drube
- O. Dunand
- P. Dusan
- L.A. Eid
- F. Emma
- M. Espino Hernandez
- M. Fila
- M. Furlano
- M. Gafencu
- M.S. Ghuysen
- M. Giani
- M. Giordano
- I. Girisgen
- N. Godefroid
- A. Godron-Dubrasquet
- I. Gojkovic
- E. Gonzalez
- I. Gökçe
- J.W. Groothoff
- S. Guarino
- A. Guffens
- P. Hansen
- J. Harambat
- S. Haumann
- G. He
- L. Heidet
- R. Helmy
- F. Hemery
- N. Hooman
- B. llanas
- A. Jankauskiene
- P. Janssens
- S. Karamaria
- I. Kazyra
- J. Koenig
- S. Krid
- P. Krug
- V. Kwon
- A. La Manna
- V. Leroy
- M. Litwin
- J. Lombet
- G. Longo
- A.C. Lungu
- A. Mallawaarachchi
- A. Marin
- P. Marzuillo
- L. Massella
- A. Mastrangelo
- H. McCarthy
- M. Miklaszewska
- A. Moczulska
- G. Montini
- A. Morawiec-Knysak
- D. Morin
- L. Murer
- I. Negru
- F. Nobili
- L. Obrycki
- H. Otoukesh
- S. Özcan
- L. Pape
- S. Papizh
- P. Parvex
- M. Pawlak-Bratkowska
- L. Prikhodina
- A. Prytula
- C. Quinlan
- A. Raes
- B. Ranchin
- N. Ranguelov
- R. Repeckiene
- C. Ronit
- R. Salomon
- R. Santagelo
- S.K. Saygılı
- S. Schaefer
- M. Schreuder
- T. Schurmans
- T. Seeman
- N. Segers
- M. Sinha
- E. Snauwaert
- B. Spasojevic
- S. Stabouli
- C. Stoica
- R. Stroescu
- E. Szczepanik
- M. Szczepańska
- K. Taranta-Janusz
- A. Teixeira
- J. Thumfart
- M. Tkaczyk
- R. Torra
- D. Torres
- N. Tram
- B. Utsch
- J. Vande Walle
- R. Vieux
- R. Vitkevic
- A. Wilhelm-Bals
- E. Wühl
- Z.Y. Yildirim
- S. Yüksel
- K. Zachwieja
- Journal volume & issue
-
Vol. 4,
no. 9
pp. 1271 – 1284
Abstract
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 childhood-diagnosed 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. Keywords: ADPKD, ADPedKD Registry, children, longitudinal
