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Kidney Week

Abstract: INFO18

AD(H)PKD: A Prospective Cohort Study on the Use of Tolvaptan in ADPKD

Session Information

Category: Genetic Diseases of the Kidneys

  • No subcategory defined

Authors

  • Grundmann, Franziska, Department II of Internal Medicine and Center for Molecular Medicine Cologne, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany
  • Mueller, Roman-Ulrich, Department II of Internal Medicine and Center for Molecular Medicine Cologne, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany
Description

Autosomal-dominant polycystic kidney disease (ADPKD) is one of the most common monogenetic diseases and leads to end stage renal disease with the requirement of renal replacement therapy in about 50% of the affected individuals by the sixth decade of life.
Many years, only supportive treatment - such as optimal blood pressure management – could be offered to the patients to delay loss of renal function. The approval of tolvaptan as the first targeted therapy of ADPKD significantly changed treatment of the disease and the counselling of patients, but real-life data on which patients are actually treated and how the therapy is managed and tolerated in daily life is still missing.
To acquire this data, we initiated the prospective AD(H)PKD cohort in 2015 which enrolls ADPKD patients who seek counselling on a therapy with tolvaptan. Therefore, the cohort contains data of patients treated with tolvaptan and of those without targeted treatment. Information including lab values, kidney volume, quality of life, adherence, genotype, extrarenal manifestation, comorbidity, side effects and complication are collected on a yearly basis. By now AD(H)PKD has enrolled more than 740 patients in 10 German centers and is successfully continuing to include patients and providing valuable data on patient characteristics, selection strategies for targeted treatment and tolerability of tolvaptan in a real-life setting. By now, it comprises one of the largest and best characterized ADPKD cohorts of the world and aims to continue until 10 years of follow up are reached. Extensive biosampling and imaging is performed in the leading center of the University of Cologne, resulting in several satellite projects on radiomics, proteomics, metabolomics and microbiome characteristics. The cohort does not only allow unique insight in the German ADPKD population, but results will be useful for guiding treatment strategies and patient counselling in the future.