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


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          Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disorder, accounting for approximately 5% of all ESRD cases worldwide. As a vasopressin receptor 2 antagonist, tolvaptan is the FDA-approved therapeutic agent for ADPKD, which is only made available to a limited number of adult patients; however, its efficacy in pediatric patients has not been reported widely. Summary: Tolvaptan was shown to delay ADPKD progression in the Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes (TEMPO) 3:4 study, Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD (REPRISE) trial, and other clinical studies. In addition to its effects on aquaretic adverse events and alanine aminotransferase elevation, the effect of tolvaptan on ADPKD is clear, sustained, and cumulative. While ADPKD is a progressive disease, the early intervention has been shown to be important and beneficial in hypotheses as well as in trials. The use of tolvaptan in pediatric ADPKD involves the following challenges: patient assessment, quality of life assessment, cost-effectiveness, safety, and tolerability. The ongoing, phase 3b, 2-part study (ClinicalTrials.gov identifier: NCT02964273) on the evaluation of tolvaptan in pediatric ADPKD (patients aged 12–17 years) may help obtain some insights. Key Messages: This review focuses on the rationality of tolvaptan use in pediatric patients with ADPKD, the associated challenges, and the suggested therapeutic approaches.

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          Most cited references54

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          Comparison of phenotypes of polycystic kidney disease types 1 and 2

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            Autosomal dominant polycystic kidney disease: the changing face of clinical management.

            Autosomal dominant polycystic kidney disease is the most common inherited kidney disease and accounts for 7-10% of all patients on renal replacement therapy worldwide. Although first reported 500 years ago, this disorder is still regarded as untreatable and its pathogenesis is poorly understood despite much study. During the past 40 years, however, remarkable advances have transformed our understanding of how the disease develops and have led to rapid changes in diagnosis, prognosis, and treatment, especially during the past decade. This Review will summarise the key findings, highlight recent developments, and look ahead to the changes in clinical practice that will likely arise from the adoption of a new management framework for this major kidney disease.
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              Magnetic resonance imaging of kidney and cyst volume in children with ADPKD.

              Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease and has important clinical manifestations in childhood. Numerous studies have documented the superiority of magnetic resonance imaging (MRI) for serial monitoring of kidney and cyst volume in this condition in adults. However, no studies have examined the utility of MRI for serial assessment of kidney and cyst volume in children with ADPKD.

                Author and article information

                Kidney Diseases
                S. Karger AG
                September 2021
                02 July 2021
                : 7
                : 5
                : 343-349
                Department of Nephrology, National Clinical Research Center for Child Health, National Children’s Regional Medical Center, The Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
                517186 Kidney Dis 2021;7:343–349
                © 2021 The Author(s) Published by S. Karger AG, Basel

                This article is licensed under the Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC). Usage and distribution for commercial purposes requires written permission. Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. Disclaimer: The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publishers and the editor(s). The appearance of advertisements or/and product references in the publication is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.

                : 27 February 2021
                : 08 April 2021
                Page count
                Figures: 1, Tables: 1, Pages: 7
                Review Article

                Cardiovascular Medicine,Nephrology
                Tolvaptan,Autosomal dominant polycystic kidney disease,Children,Therapy


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