title: Building a network of ADPKD reference centres across Europe: the EuroCYST initiative.
abstract: BACKGROUND
  Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic inherited kidney disease, affecting an estimated 600 000 individuals in Europe. The disease is characterized by age-dependent development of a multiple cysts in the kidneys, ultimately leading to end-stage renal failure and the need of renal replacement therapy in the majority of patients, typically by the fifth or sixth decade of life. The variable disease course, even within the same family, remains largely unexplained. Similarly, assessing disease severity and prognosis in an individual with ADPKD remains difficult. Epidemiological studies are limited due to the fragmentation of ADPKD research in Europe.
  
  
  METHODS
  The EuroCYST initiative aims: (i) to harmonize and develop common standards for ADPKD research by starting a collaborative effort to build a network of ADPKD reference centres across Europe and (ii) to establish a multicentric observational cohort of ADPKD patients. This cohort will be used to study factors influencing the rate of disease progression, disease modifiers, disease stage-specific morbidity and mortality, health economic issues and to identify predictive disease progression markers. Overall, 1100 patients will be enrolled in 14 study sites across Europe. Patients will be prospectively followed for at least 3 years. Eligible patients will not have participated in a pharmaceutical clinical trial 1 year before enrollment, have clinically proven ADPKD, an estimated glomerular filtration rate (eGFR) of 30 mL/min/1.73 m(2) and above, and be able to provide written informed consent. The baseline visit will include a physical examination and collection of blood, urine and DNA for biomarker and genetic studies. In addition, all participants will be asked to complete questionnaires detailing self-reported health status, quality of life, socioeconomic status, health-care use and reproductive planning. All subjects will undergo annual follow-up. A magnetic resonance imaging (MRI) scan will be carried out at baseline, and patients are encouraged to undergo a second MRI at 3-year follow-up for qualitative and quantitative kidney and liver assessments.
  
  
  CONCLUSIONS
  The ADPKD reference centre network across Europe and the observational cohort study will enable European ADPKD researchers to gain insights into the natural history, heterogeneity and associated complications of the disease as well as how it affects the lives of patients across Europe.
authors.affiliation: Institute of Physiology and Zurich Center for Integrative Human Physiology, University of Zurich, Zurich, Switzerland.
authors.forename: Katja
authors.initials: K
authors.lastname: Petzold
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: Kidney Genetics Group, Academic Nephrology Unit, Department of Infection and Immunity, University of Sheffield Medical School, Sheffield, UK.
authors.forename: Albert C M
authors.initials: AC
authors.lastname: Ong
authors.affiliation: Institute of Physiology and Zurich Center for Integrative Human Physiology, University of Zurich, Zurich, Switzerland.
authors.forename: Olivier
authors.initials: O
authors.lastname: Devuyst
authors.affiliation: Institute of Physiology and Zurich Center for Integrative Human Physiology, University of Zurich, Zurich, Switzerland.
authors.forename: Laura
authors.initials: L
authors.lastname: Rotar
authors.affiliation: Department of Nephrology and Hypertension, Friedrich-Alexander-Universität Erlangen-Nürnberg, Erlangen, Germany.
authors.forename: Kai-Uwe
authors.initials: KU
authors.lastname: Eckardt
authors.affiliation: Department of Internal Medicine IV, University Medical Center Freiburg, Freiburg, Germany.
authors.forename: Anna
authors.initials: A
authors.lastname: Köttgen
authors.affiliation: Department of Nephrology, Cliniques Universitaires Saint-Luc, Brussel, Belgium.
authors.forename: Yves
authors.initials: Y
authors.lastname: Pirson
authors.affiliation: IRCCS-Istituto di Ricerche Farmacologiche Mario Negri, Centro Anna Maria Astori, Bergamo, Italy and Unit of Nephrology, Dialysis and Transplantation, A.O. Papa Giovanni XXIII, Bergamo, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Remuzzi
authors.affiliation: Academic Department of Medical Genetics, University of Cambridge, Cambridge, UK.
authors.forename: Richard
authors.initials: R
authors.lastname: Sandford
authors.affiliation: Department of Nephrology, 1st Faculty of Medicine, Charles University, Prague, Czech Republic.
authors.forename: Vladimir
authors.initials: V
authors.lastname: Tesar
authors.affiliation: Department of Nephrology, Istanbul School of Medicine, Istanbul, Turkey.
authors.forename: Tevfik
authors.initials: T
authors.lastname: Ecder
authors.affiliation: Service de nephrologie et immunologie clinique, centre de reference des maladies renales rares (SORARE), CHU de Toulouse, universite' de Toulouse III, hopital Rangueil.
authors.forename: Dominique
authors.initials: D
authors.lastname: Chaveau
authors.affiliation: Inherited Kidney Diseases, Nephrology Department, Fundació Puigvert, Instituto de Investigaciones Biomédicas Sant Pau (IIB-Sant Pau), Universitat Autònoma de Barcelona, REDinREN, Instituto de Investigación Carlos III, Barcelona, Spain.
authors.forename: Roser
authors.initials: R
authors.lastname: Torra
authors.affiliation: Department of Nephrology, Charité Campus Mitte, Charité Universitätsmedizin Berlin, Berlin, Germany.
authors.forename: Klemens
authors.initials: K
authors.lastname: Budde
authors.affiliation: Department of Nephrology, Centre Hospitalier Universitaire de Brest, Brest, France.
authors.forename: Yannick
authors.initials: Y
authors.lastname: Le Meur
authors.affiliation: Division of Nephrology, University Hospital Zurich, Zurich, Switzerland.
authors.forename: Rudolf P
authors.initials: RP
authors.lastname: Wüthrich
authors.affiliation: Institute of Physiology and Zurich Center for Integrative Human Physiology, University of Zurich, Zurich, Switzerland Division of Nephrology, University Hospital Zurich, Zurich, Switzerland.
authors.forename: Andreas L
authors.initials: AL
authors.lastname: Serra
mesh_terms: D015415:Biomarkers / Q000032:analysis; D005060:Europe; D005919:Glomerular Filtration Rate; D006296:Health Services; D006304:Health Status; D006801:Humans; D008137:Longitudinal Studies; D008279:Magnetic Resonance Imaging; D016891:Polycystic Kidney, Autosomal Dominant / Q000503:physiopathology / Q000628:therapy*; D011379:Prognosis; D012017:Referral and Consultation; D012107:Research Design; D059039:Standard of Care / Q000458:organization & administration*; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D015415:Biomarkers
keywords: ADPKD; EuroCYST; biomarker; cohort; risk factors