Building a network of ADPKD reference centres across Europe: the EuroCYST initiative
Por:
Petzold, K, Gansevoort, RT, Ong, ACM, Devuyst, O, Rotar, L, Eckardt, KU, Kottgen, A, Pirson, Y, Remuzzi, G, Sandford, R, Tesar, V, Ecder, T, Chaveau, D, Torra, R, Budde, K, Le Meur, Y, Wuthrich, RP, Serra, AL
Publicada:
1 sep 2014
Resumen:
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 enrolment, 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.
Filiaciones:
Petzold, K:
Univ Zurich, Inst Physiol, Zurich, Switzerland
Univ Zurich, Zurich Ctr Integrat Human Physiol, Zurich, Switzerland
Gansevoort, RT:
Univ Groningen, Univ Med Ctr Groningen, Dept Nephrol, Groningen, Netherlands
Ong, ACM:
Univ Sheffield, Sch Med, Dept Infect & Immun, Kidney Genet Grp,Acad Nephrol Unit, Sheffield, S Yorkshire, England
Devuyst, O:
Univ Zurich, Inst Physiol, Zurich, Switzerland
Univ Zurich, Zurich Ctr Integrat Human Physiol, Zurich, Switzerland
Rotar, L:
Univ Zurich, Inst Physiol, Zurich, Switzerland
Univ Zurich, Zurich Ctr Integrat Human Physiol, Zurich, Switzerland
Eckardt, KU:
Univ Erlangen Nurnberg, Dept Nephrol & Hypertens, D-91054 Erlangen, Germany
Kottgen, A:
Univ Med Ctr Freiburg, Dept Internal Med 4, Freiburg, Germany
Pirson, Y:
Clin Univ St Luc, Dept Nephrol, Brussels, Belgium
Remuzzi, G:
Ctr Anna Maria Astori, IRCCS Ist Ric Farmacol Mario Negri, Bergamo, Italy
AO Papa Giovanni XXIII, Unit Nephrol Dialysis & Transplantat, Bergamo, Italy
Sandford, R:
Univ Cambridge, Acad Dept Med Genet, Cambridge, England
Tesar, V:
Charles Univ Prague, Fac Med 1, Dept Nephrol, Prague, Czech Republic
Ecder, T:
Istanbul Sch Med, Dept Nephrol, Istanbul, Turkey
Chaveau, D:
Univ Toulouse 3, Hop Rangueil, CHU Toulouse, Serv Nephrol & Immunol Clin,Ctr Reference Malad R, F-31062 Toulouse, France
Torra, R:
Univ Autonoma Barcelona, Inst Invest Carlos III, IIB St Pau, Nephrol Dept,Fundacio Puigvert,REDinREN, E-08193 Barcelona, Spain
Budde, K:
Charite, Dept Nephrol, D-13353 Berlin, Germany
Le Meur, Y:
CHU Brest, Dept Nephrol, F-29285 Brest, France
Wuthrich, RP:
Univ Zurich Hosp, Div Nephrol, CH-8091 Zurich, Switzerland
Serra, AL:
Univ Zurich, Inst Physiol, Zurich, Switzerland
Univ Zurich, Zurich Ctr Integrat Human Physiol, Zurich, Switzerland
Univ Zurich Hosp, Div Nephrol, CH-8091 Zurich, Switzerland
Green Published, Green Accepted, Bronze
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