Building a network of ADPKD reference centres across Europe: the EuroCYST initiative
Jazyk angličtina Země Anglie, Velká Británie Médium print
Typ dokumentu časopisecké články, pozorovací studie, práce podpořená grantem
PubMed
25165183
DOI
10.1093/ndt/gfu091
PII: gfu091
Knihovny.cz E-zdroje
- Klíčová slova
- ADPKD, EuroCYST, biomarker, cohort, risk factors,
- MeSH
- biologické markery analýza MeSH
- hodnoty glomerulární filtrace MeSH
- konziliární vyšetření a konzultace * MeSH
- lidé MeSH
- longitudinální studie MeSH
- magnetická rezonanční tomografie MeSH
- mladý dospělý MeSH
- polycystické ledviny autozomálně dominantní patofyziologie terapie MeSH
- prognóza MeSH
- průzkumy a dotazníky MeSH
- standardní péče organizace a řízení MeSH
- výzkumný projekt * MeSH
- zdravotní stav MeSH
- zdravotnické služby * MeSH
- Check Tag
- lidé MeSH
- mladý dospělý MeSH
- Publikační typ
- časopisecké články MeSH
- pozorovací studie MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Evropa MeSH
- Názvy látek
- biologické markery MeSH
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.
Academic Department of Medical Genetics University of Cambridge Cambridge UK
Department of Internal Medicine 4 University Medical Center Freiburg Freiburg Germany
Department of Nephrology 1st Faculty of Medicine Charles University Prague Czech Republic
Department of Nephrology Centre Hospitalier Universitaire de Brest Brest France
Department of Nephrology Charité Campus Mitte Charité Universitätsmedizin Berlin Berlin Germany
Department of Nephrology Cliniques Universitaires Saint Luc Brussel Belgium
Department of Nephrology Istanbul School of Medicine Istanbul Turkey
Division of Nephrology University Hospital Zurich Zurich Switzerland
Nephrol Dial Transplant. 2014 Dec;29(12):2353 PubMed
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