Ron T. Gansevoort 1 , Mustafa Arici 2 , Thomas Benzing 3 , Henrik Birn 4 , 5 , Giovambattista Capasso 6 , Adrian Covic 7 , Olivier Devuyst 8 , 9 , Christiane Drechsler 10 , Kai-Uwe Eckardt 11 , Francesco Emma 12 , Bertrand Knebelmann 13 , Yannick Le Meur 14 , Ziad A. Massy 15 , 16 , 17 , Albert C.M. Ong 18 , Alberto Ortiz 19 , Franz Schaefer 20 , Roser Torra 21 , 22 , Raymond Vanholder 23 , Andrzej Więcek 24 , Carmine Zoccali 25 , Wim Van Biesen 23
29 January 2016
Recently, the European Medicines Agency approved the use of the vasopressin V2 receptor antagonist tolvaptan to slow the progression of cyst development and renal insufficiency of autosomal dominant polycystic kidney disease (ADPKD) in adult patients with chronic kidney disease stages 1–3 at initiation of treatment with evidence of rapidly progressing disease. In this paper, on behalf of the ERA-EDTA Working Groups of Inherited Kidney Disorders and European Renal Best Practice, we aim to provide guidance for making the decision as to which ADPKD patients to treat with tolvaptan. The present position statement includes a series of recommendations resulting in a hierarchical decision algorithm that encompasses a sequence of risk-factor assessments in a descending order of reliability. By examining the best-validated markers first, we aim to identify ADPKD patients who have documented rapid disease progression or are likely to have rapid disease progression. We believe that this procedure offers the best opportunity to select patients who are most likely to benefit from tolvaptan, thus improving the benefit-to-risk ratio and cost-effectiveness of this treatment. It is important to emphasize that the decision to initiate treatment requires the consideration of many factors besides eligibility, such as contraindications, potential adverse events, as well as patient motivation and lifestyle factors, and requires shared decision-making with the patient.