Directly to content
  1. Publishing |
  2. Search |
  3. Browse |
  4. Recent items rss |
  5. Open Access |
  6. Jur. Issues |
  7. DeutschClear Cookie - decide language by browser settings

Rationale, design and objectives of ARegPKD, a European ARPKD registry study

Ebner, Kathrin ; Feldkötter, Markus ; Ariceta, Gema ; Bergmann, Carsten ; Büttner, Reinhard ; Doyon, Anke ; Duzova, Ali ; Göbel, Heike ; Haffner, Dieter ; Hero, Barbara ; Hoppe, Bernd ; Illig, Thomas ; Jankauskiene, Augustina ; Klopp, Norman ; König, Jens ; Litwin, Mieczyslaw ; Mekahli, Djalila ; Ranchin, Bruno ; Sander, Anja ; Testa, Sara ; Weber, Lutz Thorsten ; Wicher, Dorota ; Yuzbasioglu, Ayse ; Zerres, Klaus ; Dötsch, Jörg ; Schaefer, Franz ; Liebau, Max Christoph

In: BMC nephrology, 16 (2015), Nr. 22. pp. 1-7. ISSN 1471-2369

[img]
Preview
PDF, English
Download (517kB) | Lizenz: Creative Commons LizenzvertragRationale, design and objectives of ARegPKD, a European ARPKD registry study by Ebner, Kathrin ; Feldkötter, Markus ; Ariceta, Gema ; Bergmann, Carsten ; Büttner, Reinhard ; Doyon, Anke ; Duzova, Ali ; Göbel, Heike ; Haffner, Dieter ; Hero, Barbara ; Hoppe, Bernd ; Illig, Thomas ; Jankauskiene, Augustina ; Klopp, Norman ; König, Jens ; Litwin, Mieczyslaw ; Mekahli, Djalila ; Ranchin, Bruno ; Sander, Anja ; Testa, Sara ; Weber, Lutz Thorsten ; Wicher, Dorota ; Yuzbasioglu, Ayse ; Zerres, Klaus ; Dötsch, Jörg ; Schaefer, Franz ; Liebau, Max Christoph underlies the terms of Creative Commons Attribution 3.0 Germany

Citation of documents: Please do not cite the URL that is displayed in your browser location input, instead use the DOI, URN or the persistent URL below, as we can guarantee their long-time accessibility.

Abstract

Background: Autosomal recessive polycystic kidney disease (ARPKD) is a rare but frequently severe disorder that is typically characterized by cystic kidneys and congenital hepatic fibrosis but displays pronounced phenotypic heterogeneity. ARPKD is among the most important causes for pediatric end stage renal disease and a leading reason for liver-, kidney- or combined liver kidney transplantation in childhood. The underlying pathophysiology, the mechanisms resulting in the observed clinical heterogeneity and the long-term clinical evolution of patients remain poorly understood. Current treatment approaches continue to be largely symptomatic and opinion-based even in most-advanced medical centers. While large clinical trials for the frequent and mostly adult onset autosomal dominant polycystic kidney diseases have recently been conducted, therapeutic initiatives for ARPKD are facing the challenge of small and clinically variable cohorts for which reliable end points are hard to establish. Methods/Design: ARegPKD is an international, mostly European, observational study to deeply phenotype ARPKD patients in a pro- and retrospective fashion. This registry study is conducted with the support of the German Society for Pediatric Nephrology (GPN) and the European Study Consortium for Chronic Kidney Disorders Affecting Pediatric Patients (ESCAPE Network). ARegPKD clinically characterizes long-term ARPKD courses by a web-based approach that uses detailed basic data questionnaires in combination with yearly follow-up visits. Clinical data collection is accompanied by associated biobanking and reference histology, thus setting roots for future translational research. Discussion: The novel registry study ARegPKD aims to characterize miscellaneous subcohorts and to compare the applied treatment options in a large cohort of deeply characterized patients. ARegPKD will thus provide evidence base for clinical treatment decisions and contribute to the pathophysiological understanding of this severe inherited disorder.

Item Type: Article
Journal or Publication Title: BMC nephrology
Volume: 16
Number: 22
Publisher: BioMed Central
Place of Publication: London
Date Deposited: 09 Dec 2015 08:36
Date: 2015
ISSN: 1471-2369
Page Range: pp. 1-7
Faculties / Institutes: Medizinische Fakultät Heidelberg > Universitätskinderklinik
Medizinische Fakultät Heidelberg > Institut für Medizinische Biometrie und Informatik
Subjects: 610 Medical sciences Medicine
About | FAQ | Contact | Imprint |
OA-LogoDINI certificate 2013Logo der Open-Archives-Initiative