title: Recommendations for the use of tolvaptan in autosomal dominant polycystic kidney disease: a position statement on behalf of the ERA-EDTA Working Groups on Inherited Kidney Disorders and European Renal Best Practice.
abstract: 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.
authors.affiliation: Department of Nephrology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Ron T
authors.initials: RT
authors.lastname: Gansevoort
authors.affiliation: Department of Nephrology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
authors.forename: Mustafa
authors.initials: M
authors.lastname: Arici
authors.affiliation: Department II of Internal Medicine and Centre for Molecular Medicine Cologne, University of Cologne, Cologne, Germany.
authors.forename: Thomas
authors.initials: T
authors.lastname: Benzing
authors.affiliation: Department of Renal Medicine, Aarhus University Hospital, Aarhus, Denmark Department of Biomedicine, Aarhus University, Aarhus, Denmark.
authors.forename: Henrik
authors.initials: H
authors.lastname: Birn
authors.affiliation: Department of Nephrology, Second University of Naples, Naples, Italy.
authors.forename: Giovambattista
authors.initials: G
authors.lastname: Capasso
authors.affiliation: Nephrology Clinic, Dialysis and Renal Transplant Center, 'C.I. PARHON' University Hospital, and 'Grigore T. Popa' University of Medicine, Iasi, Romania.
authors.forename: Adrian
authors.initials: A
authors.lastname: Covic
authors.affiliation: Institute of Physiology, University of Zurich, Zurich, Switzerland Division of Nephrology, UCL Medical School, Brussels, Belgium.
authors.forename: Olivier
authors.initials: O
authors.lastname: Devuyst
authors.affiliation: Renal Division, University of Würzburg, University Hospital, Wurzburg, Germany.
authors.forename: Christiane
authors.initials: C
authors.lastname: Drechsler
authors.affiliation: Department of Nephrology and Hypertension, Friedrich-Alexander University Erlangen-Nürnberg (FAU), Erlangen, Germany.
authors.forename: Kai-Uwe
authors.initials: KU
authors.lastname: Eckardt
authors.affiliation: Department of Nephrology and Urology, Bambino Gesù Children's Hospital-IRCCS, Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Emma
authors.affiliation: Department of Nephrology, Hôpital Necker, Paris Descartes University, Paris, France.
authors.forename: Bertrand
authors.initials: B
authors.lastname: Knebelmann
authors.affiliation: Service de Néphrologie, Hémodialyse et Transplantation Rénale, Hôpital La Cavale Blanche, Centre Hospitalier Régional Universitaire de Brest, Brest, France.
authors.forename: Yannick
authors.initials: Y
authors.lastname: Le Meur
authors.affiliation: Division of Nephrology, Ambroise Paré Hospital, Assistance Publique Hôpitaux de Paris, Boulogne-Billancourt/Paris, France Inserm U-1018, Equipe 5, Villejuif, France University of Paris Saclay and Paris Ouest-Versailles-Saint-Quentin-en-Yvelines (UVSQ), France.
authors.forename: Ziad A
authors.initials: ZA
authors.lastname: Massy
authors.affiliation: Academic Nephrology Unit, University of Sheffield Medical School, Sheffield, UK.
authors.forename: Albert C M
authors.initials: AC
authors.lastname: Ong
authors.affiliation: IIS-Fundacion Jimenez Diaz-UAM and REDINREN, Madrid, Spain.
authors.forename: Alberto
authors.initials: A
authors.lastname: Ortiz
authors.affiliation: Pediatric Nephrology Division, Center for Pediatrics and Adolescent Medicine, Heidelberg University Hospital, Heidelberg, Germany.
authors.forename: Franz
authors.initials: F
authors.lastname: Schaefer
authors.affiliation: Inherited Kidney Diseases Nephrology Department, Fundació Puigvert Instituto de Investigaciones Biomédicas Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain REDINREN, Barcelona, Spain.
authors.forename: Roser
authors.initials: R
authors.lastname: Torra
authors.affiliation: Renal Division, Ghent University Hospital, Ghent, Belgium.
authors.forename: Raymond
authors.initials: R
authors.lastname: Vanholder
authors.affiliation: Department of Nephrology, Transplantation and Internal Medicine, Medical University of Silesia in Katowice, Katowice, Poland.
authors.forename: Andrzej
authors.initials: A
authors.lastname: Więcek
authors.affiliation: CNR-IFC Clinical Epidemiology and Pathophysiology of Renal Diseases and Hypertension Unit, Reggio Calabria c/o Ospedali Riuniti, Reggio Calabria, Italy.
authors.forename: Carmine
authors.initials: C
authors.lastname: Zoccali
authors.affiliation: Renal Division, Ghent University Hospital, Ghent, Belgium.
authors.forename: Wim
authors.initials: W
authors.lastname: Van Biesen
mesh_terms: D000328:Adult; D065092:Antidiuretic Hormone Receptor Antagonists / Q000627:therapeutic use; D001552:Benzazepines / Q000627:therapeutic use*; D018450:Disease Progression; D005060:Europe; D006801:Humans; D007010:Hyponatremia; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000235:genetics*; D012955:Societies, Medical; D000077602:Tolvaptan
chemical_list: D065092:Antidiuretic Hormone Receptor Antagonists; D001552:Benzazepines; D000077602:Tolvaptan
keywords: ADPKD; tolvaptan; vasopressin V2 receptor antagonist