Title

ADPedKD: A Global Online Platform on the Management of Children With ADPKD.

Document Type

Journal Article

Publication Date

9-1-2019

Journal

Kidney Int Rep

Volume

4

Issue

9

DOI

10.1016/j.ekir.2019.05.015

Grant Information

CTSI FUNDED.

Funding text #1 DM is supported by the Clinical Research Fund of UZ Leuven . LGW is supported by the UAB Hepatorenal Fibrocystic Disease Core Center ( National Institutes of Health (NIH)/ National Institute of Diabetes and Digestive and Kidney Diseases P30 DK074038 ), the Polycystic Kidney Disease Foundation, and the Clinical and Translational Science Institute at Children’s National (CTSI-CN; NIH/ National Center for Advancing Translational Sciences UL1TR001876 ). MCL is supported by the Koeln Fortune program and the GEROK program of the Medical Faculty of University of Cologne. SDR is supported by the Fund for Scientific Research, Flanders 11M5214N .

Funding text #2 To date, ADPedKD has been supported by an initiation award from the University Hospitals Leuven coordinating center, as well as a research grant from the European Society of Pediatric Nephrology and the European Renal Association–European Dialysis and Transplant Association. The funding agencies had no role in study design, the collection, analysis, and interpretation of data, or in the manuscript preparation.

Funding text #3 The authors would like to thank the German Society for Pediatric Nephrology (GPN) for their endorsement; ‘VZW Bas, Stoere strijder’ for financial support of the project; and Joanna De Vis, Veerle Verbeek and Helga Wielandt for data entry of the Leuven patient cohort. The authors would like to thank the patients enrolled in the ADPedKD registry and their families.

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.

Peer Reviewed

1

Open Access

1

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