ADPedKD: A global online platform on the management of children with ADPKD

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.

C1 [De Rechter, Stephanie; Mekahli, Djalila] Univ Hosp Leuven, Dept Pediat Nephrol, Herestr 49, B-3000 Leuven, Belgium.

[De Rechter, Stephanie; Mekahli, Djalila] Katholieke Univ Leuven, Dept Dev & Regenerat, PKD Res Grp, Leuven, Belgium.

[Bockenhauer, Detlef] UCL Ctr Nephrol, London, England.

[Bockenhauer, Detlef] Great Ormond St Hosp NHS Fdn Trust, London, England.

[Guay-Woodford, Lisa M.] Childrens Natl Hlth Syst, Ctr Translat Sci, Washington, DC USA.

[Liu, Isaac] Natl Univ Hlth Syst, Khoo Teck Puat Natl Univ, Childrens Med Inst, Singapore, Singapore.

[Mallett, Andrew J.] Royal Brisbane & Womens Hosp, Kidney Hlth Serv, Brisbane, Qld, Australia.

[Mallett, Andrew J.] Royal Brisbane & Womens Hosp, Conjoint Renal Res Lab, Brisbane, Qld, Australia.

[Mallett, Andrew J.] Univ Queensland, Fac Med, Brisbane, Qld, Australia.

[Mallett, Andrew J.] Univ Queensland, Inst Mol Biosci, Brisbane, Qld, Australia.

[Mallett, Andrew J.] KidGen Collaborat & Australian Genom Hlth Allianc, Melbourne, Vic, Australia.

[Soliman, Neveen A.] Cairo Univ, Ctr Pediat Nephrol & Transplantat, Kasr Al Ainy Sch Med, Dept Pediat, Cairo, Egypt.

[Sylvestre, Lucimary C.] Hosp Pequeno Principe, Curitiba, Parana, Brazil.

[Schaefer, Franz] Heidelberg Univ, Ctr Pediat & Adolescent Med, Div Pediat Nephrol, Med Ctr, Heidelberg, Germany.

[Liebau, Max C.] Univ Hosp Cologne, Dept Pediat, Cologne, Germany.

[Liebau, Max C.] Univ Hosp Cologne, Ctr Mol Med, Cologne, Germany.

[Adamczyk, P.; Bjanid, O.; Brylka, A.; Morawiec-Knysak, A.; Szczepanska, M.] Dept Pediat, Zabrze, Poland.

[Akinci, N.] Sariyer SISLI Hamidiye Etfal Res & Educ Hosp, Istanbul, Turkey.

[Alpay, H.; Cicek, N.; Gokce, I] Marmara Univ, Sch Med, Div Pediat Nephrol, Istanbul, Turkey.

[Ardelean, C.; Chirita, A.; Gafencu, M.; Stroescu, R.] Timisoara Children Hosp, Timisoara, Romania.

[Ayasreh, N.; Furlano, M.; Torra, R.] Fundacio Puigvert, Barcelona, Spain.

[Aydin, Z.; Bayrakci, U. S.] Ankara Univ Hlth Sci, Child Hlth & Dis, Ankara, Turkey.

[Bael, A.; Docx, M.; Segers, N.] Koningin Paola Kinderziekenhuis Antwerpen, Antwerp, Belgium.

[Baudouin, V; Cambier, A.; Couderc, A.; Dossier, C.; Kwon, V] Hop Robert Debre, AP HP, Paris, France.

[Bensman, A.; Biebuyck, A.; Boyer, O.; Charbit, M.; Heidet, L.; Krid, S.; Krug, P.; Salomon, R.] Pediat Nephrol Necker Hosp, Paris, France.

[Bialkevich, H.; Kazyra, I] 2nd City Childrens Clin Hosp, Natl Ctr Pediat Nephrol & RRT, Minsk, BELARUS.

[Caliskan, S.; Ozcan, S.; Saygili, S. K.] Istanbul Cerrahpasa Fac Med, Istanbul, Turkey.

[Camelio, A.; Nobili, F.; Vieux, R.] CHU Besancon, Besancon, France.

[Carbone, V; Diomeda, F.; Torres, D.] Pediat Nephrol Unit Bari, Bari, Italy.

[Chiodini, B.] HUDERF, Brussels, Belgium.

[Collard, L.] CHR La Citadelle, Liege, Belgium.

[Conceicao, M.; Teixeira, A.] Ctr Hosp Porto, Ctr Materno Infantil Norte, Porto, Portugal.

[Constantinescu, I; Lungu, A. C.; Marin, A.; Negru, I; Stroescu, R.] Fundeni Clin Inst, Bucharest, Romania.

[Crapella, B.; Giani, M.; Mastrangelo, A.; Montini, G.] Fdn IRCCS Ca Granda, Pediat Nephrol Dialysis & Transplant Unit, Milan, Italy.

[Cvetkovic, M.; Gojkovic, I] Univ Childrens Hosp, Belgrade, Serbia.

[Dima, B.] Clin Europe Hop St Elisabeth, Brussels, Belgium.

[Dolan, N.] Our Ladys Childrens Hosp, Dublin, Ireland.

[Drozdz, D.; Miklaszewska, M.; Zachwieja, K.] Jagiellonian Univ, Med Coll Cracow, Pediat Nephrol & Hypertens, Krakow, Poland.

[Drube, J.; Pape, L.] Hannover Med Sch, Hannover, Germany.

[Dunand, O.; Leroy, V] Pediat Nephrol Unit St Denis, St Denis, Reunion, France.

[Dusan, P.; Spasojevic, B.; Stabouli, S.] Aristotle Univ Thessaloniki, Dept Pediat, Thessaloniki, Greece.

[Eid, L. A.] Dubai Hosp, Pediat Nephrol Dept, Dubai, U Arab Emirates.

[Emma, F.; Massella, L.] Bambino Gesu Pediat Hosp, Rome, Italy.

[Espino Hernandez, M.] Hosp Infantil 12 Octubre Madrid, Madrid, Spain.

[Fila, M.; Hemery, F.; Morin, D.] CHU Arnaud Villeneuve, Montpellier, France.

[Ghuysen, Ms] CHU Liege, Liege, Belgium.

[Giordano, M.] Pediat Nephrol Unit, Bari, Italy.

[Girisgen, I; Yuksel, S.] Pamukkale Univ, Med Fac, Dept Pediat Nephrol, Denizli, Turkey.

[Godefroid, N.; Ranguelov, N.] Clin Univ St Luc, Brussels, Belgium.

[Godron-Dubrasquet, A.; Harambat, J.; Ilanas, B.] Bordeaux Univ Childrens Hosp, Bordeaux, France.

[Gonzalez, E.] Childrens Univ Hosp, Geneva, Switzerland.

[Groothoff, J. W.] Emma Childrens Hosp, Amsterdam, Netherlands.

[Guarino, S.; La Manna, A.; Marzuillo, P.] Univ Campania Luigi Vanvitelli, Caserta, Italy.

[Guffens, A.] CHC Clin Esperence, Montegnee, Belgium.

[Hansen, P.] CHU Tivoli, La Louviere, Belgium.

[Haumann, S.] Univ Klinikum Koln, Cologne, Germany.

[He, G.] Foshan Women & Children Hosp, Foshan, Peoples R China.

[Helmy, R.] Cairo Univ, Kasr Al Ainy Sch Med, Cairo, Egypt.

[Hooman, N.; Otoukesh, H.] Iran Univ Med Sci, Aliasghar Clin Res Dev Unit, Tehran, Iran.

[Janssens, P.] Univ Hosp Brussels, Brussels, Belgium.

[Karamaria, S.; Prytula, A.; Raes, A.; Snauwaert, E.; Vande Walle, J.] UZ Gent, Ghent, Belgium.

[Koenig, J.] Univ Hosp Muenster, Munster, Germany.

[Litwin, M.; Obrycki, L.] Childrens Mem Hlth Inst, Warsaw, Poland.

[Lombet, J.] CHR Citadelle, Liege, Belgium.

[Longo, G.; Murer, L.] Hosp Univ Padova, Pediat Nephrol Dialysis & Transplant Unit, Padua, Italy.

[Mallawaarachchi, A.] Garvan Inst, Darlinghurst, NSW, Australia.

[Mallawaarachchi, A.] Royal Prince Alfred Hosp, Camperdown, NSW, Australia.

[Mallawaarachchi, A.; McCarthy, H.; Quinlan, C.] KidGen, Sydney, NSW, Australia.

[McCarthy, H.] Childrens Hosp Westmead, Westmead, NSW, Australia.

[McCarthy, H.] Sydney Childrens Hosp, Sydney, NSW, Australia.

[Papizh, S.; Prikhodina, L.] Pirogov Russian Nat Res Med Uni, Res & Clin Inst Pediat, Moscow, Russia.

[Parvex, P.; Wilhelm-Bals, A.] Childrens Univ Hosp Geneva, Geneva, Switzerland.

[Pawlak-Bratkowska, M.; Szczepanik, E.; Tkaczyk, M.] Polish Mothers Mem Hosp, Res Inst, Lodz, Poland.

[Quinlan, C.] RCH Melbourne, Melbourne, Vic, Australia.

[Ranchin, B.] Hop Femme Mere Enfant, Bron, France.

[Ronit, C.] Ctr Hosp Luxembourg, Clin Pediat, Luxembourg, Luxembourg.

[Schaefer, S.; Wuehl, E.] Ctr Pediat & Adolescent, Div Pediat Nephrol, Heidelberg, Germany.

[Schreuder, M.] Radboudumc Amalia Childrens Hosp, Nijmegen, Netherlands.

[Schurmans, T.; Tram, N.] CHU Charleroi, Charleroi, Belgium.

[Seeman, T.] Charles Univ Prague, Prague, Czech Republic.

[Seeman, T.] Motol Univ Hosp, Prague, Czech Republic.

[Sinha, M.] Evelina London Childrens Hosp, London, England.

[Taranta-Janusz, K.] Dept Pediat & Nephrol, Bialystok, Poland.

[Thumfart, J.] Berlin Charite Univ Med, Berlin, Germany.

[Utsch, B.] Herford Hosp, Dept Paediat, Herford, Germany.

[Yildirim, Z. Y.] Istanbul Univ, Fac Med, Pediat Nephrol Dept, Istanbul, Turkey.