Kidney Week

Abstract: SA-PO473

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

Session Information

• ADPKD: Clinical Studies
  October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidney

• 1001 Genetic Diseases of the Kidney: Cystic

Authors

• Mueller, Roman-Ulrich, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Roman-Ulrich Mueller,

• Todorova, Polina, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Polina Todorova,

• Suárez, Victor, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Victor Suárez,

• Schermer, Bernhard, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Bernhard Schermer,

• Benzing, Thomas, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Thomas Benzing,

• Burst, Volker Rolf, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Volker Rolf Burst,

• Grundmann, Franziska, Dept. 2 of Internal Medicine, University of Cologne, Koeln, Germany

Franziska Grundmann,

Background

The approval of tolvaptan as the first targeted therapy of autosomal-dominant polycystic kidney disease is a significant progress in the treatment of this disease. But how is this therapy managed in the real-life setting? How is it accepted by patients taking into account the consecutive polyuria?

Methods

To answer these questions, we initiated the AD(H)PKD registry which enrolls ADPKD patients that have not reached ESRD yet and present with the question whether tolvaptan would be a treatment option. We collect data on a yearly basis including lab values, kidney volume, quality of life, adherence, genotype, extrarenal manifestations, comorbidity, side effects and complications.

Results

Since the start of the study at the end of 2015 until May 2018 more than 450 patients could be enrolled. Here, we present - apart from the general characterization of the cohort – first data regarding the use of tolvaptan in the real-life setting. The urine volume increases to 5-7.5 liters in the majority of patients on tolvaptan. The largest increase occurs when starting the first dose step (45/15 mg), whilst the further uptitration (60/30 and 90/30 mg) only shows a minor additional effect on urine output. The same holds true for urine osmolality. Adherence to the therapy is currently about 80%; most patients that discontinue tolvaptan do so early after initation with polyuria-related events being the most common reason. More than 75% of patients report little to no problems with the therapy in everyday life. Patients skip a dose 1-2x per month on average and do so mainly due to professional and leisure-time activities.

Conclusion

The AD(H)PKD study continues successful enrolment and allows for a first insight into patient-reported outcomes like quality of life and feasibility of the therapy. The adherence of about 80% confirms the data obtained in TEMPO 3:4. Furthermore, AD(H)PKD provides the first systematic data on urine volume and osmolality in the real-life setting. These results will be very helpful for guiding management of ADPKD patients and patient counselling in the future.

Funding

• Commercial Support –