An update on the use of tolvaptan for ADPKD: Consensus statement on behalf of the ERA Working Group on Inherited Kidney Disorders (WGIKD), the European Rare Kidney Disease Reference Network (ERKNet) and Polycystic Kidney Disease International (PKD-International)

Abstract

Abstract The approval of the vasopressin V2-receptor antagonist tolvaptan – based on the landmark TEMPO 3:4 trial – has marked a transformation in the management of autosomal dominant polycystic kidney disease (ADPKD). This development has advanced patient care in ADPKD from general measures to prevent progression of chronic kidney disease to targeting disease specific mechanisms. However, considering the long-term nature of this treatment as well as potential side effects, evidence-based approaches to initiate treatment only in patients with rapidly progressing disease are crucial. In 2016, the position statement issued by the ERA was the first society-based recommendation on the use of tolvaptan and has served as a widely-used decision-making tool for nephrologists. Since then, considerable practical experience regarding the use of tolvaptan in ADPKD has accumulated. More importantly, additional data from REPRISE, a second randomized clinical trial (RCT) examining the use of tolvaptan in later stage disease has added important evidence to the field, as have post-hoc studies of these RCTs. To incorporate this new knowledge, we provide an updated algorithm to guide patient selection for treatment with tolvaptan and add practical advice for its use.