Abstract: FR-PO313

First Year Follow-Up Data from the German ADPKD Tolvaptan Treatment Registry – AD(H)PKD

Session Information

Category: Genetic Diseases of the Kidney

  • 801 Cystic Kidney Diseases

Authors

  • Mueller, Roman-Ulrich, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Grundmann, Franziska, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Todorova, Polina, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Burkert, Katharina, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Witte, Claudia, University of Cologne, Cologne, Germany
  • Burst, Volker Rolf, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Persigehl, Thorsten, Department of Radiology, University Hospital Cologne, Cologne, Germany
  • Schermer, Bernhard, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
  • Benzing, Thomas, Department 2 of Internal Medicine, Renal Division; University of Cologne, Cologne, Germany
Background

Admission of Tolvaptan in Europe by the EMA as the first targeted therapy of autosomal-dominant polycystic kidney disease (ADPKD) based on the findings of the TEMPO 3:4 trial is a milestone in the treatment of this disease. After the significant benefit on eGFR in TEMPO 3:4 and 4:4 more data on this therapy regarding crucial questions in the real-life setting would be highly valuable. How do patients accept the treatment taking into account polyuria? Is the target dose reached? What side effects occur? How is the effect regarding kidney function and volume? Which patients are selected for treatment? How does Tolvaptan affect quality of life? How good is the adherence to the therapy?

Methods

In order to answer these questions we established the multicentric German AD(H)PKD registry. Patients that are generally eligible for Tolvaptan, independent of whether actually taking the drug or not, can be included in this observational study. Blood values, kidney volume from imaging data, indicators of quality of life, adherence to therapy, the actual dose administered, genotype and data regarding extrarenal manifestations, comorbidity, side effects and complications are documented. After enrolment patients are followed-up in yearly visits for ten years.

Results

We have been able to recruit more than 270 ADPKD patients so far and have started the first-year follow-up visits at the end of 2016. Consequently, analysis of this cohort allows for the first characterization of patients presented for evaluation regarding initiation of Tolvaptan on the one hand. On the other hand the first-year data provide an interesting insight into which patients were selected for treatment and sheds light on dosing strategies. Furthermore, adherence to therapy and the impact on quality of life are analyzed.

Conclusion

The AD(H)PKD-registry provides the first comprising dataset on the German cohort of ADPKD patients eligible for treatment with Tolvaptan and analyzes the selection criteria applied by German nephrologists as well as tolerability, side effects and impact on other kidney-related outcomes. Follow-up of this cohort will provide valuable data that can help in counseling patients and informing physicians dealing with this novel treatment opportunity.

Funding

  • Commercial Support