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.

Tolvaptan in Pediatric Autosomal Dominant Polycystic Kidney Disease: From Here to Where?

“…Overall, although the use of tolvaptan requires careful consideration and balancing of the benefits and risks, tolvaptan is a valuable treatment option to delay the progression of ADPKD in patients at risk of or showing signs of rapidly progressing disease. Data from the global ADPedKD projects will help improve our understanding of disease progression from early disease stages [54]. The first trial on the treatment of pediatric ADPKD using tolvaptan is currently underway, which is expected to yield encouraging results.…”

Metabolic Reprogramming and Reconstruction: Integration of Experimental and Computational Studies to Set the Path Forward in ADPKD

“…This may, as a consequence, influence their life-style. There is a significant discrepancy regarding data obtained in adults vs. children as diagnostic criteria are highly variable, leading to incomparable studies and results ( 166 ). In children with ADPKD, EU guidelines recommends to stratify high risk patients and Tolvaptan is currently being tested in EU in children with ADPKD with high risk of rapid progression ( 167 ).…”

Autosomal-dominante polyzystische NierenerkrankungManagement im Kindes- und Jugendalter

“…Mit der Unterstützung unterschiedlicher Fachgesellschaften, wie z. B. der Gesellschaft für Pädiatrische Nephrologie (GPN), wurde in den vergangenen Jahren eine internationale Beobachtungsstudie etabliert, welche die natürlichen Verläufe von Kindern mit bereits diagnostizierter ADPKD detailliert nachverfolgt [10]. Aufbauend auf ähnlichen Initiativen z.…”