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Abstract: TH-PO835

Quality of Life and Tolerability of Tolvaptan in Swiss ADPKD Patients

Session Information

Category: Genetic Diseases of the Kidneys

  • 1001 Genetic Diseases of the Kidneys: Cystic

Authors

  • Anderegg, Manuel Andreas, Inselspital, Bern University Hospital, Bern, Switzerland
  • Dhayat, Nasser, Inselspital, Bern University Hospital, Bern, Switzerland
  • Sommer, Grit, Inselspital, Bern University Hospital, Bern, Switzerland
  • Semmo, Mariam, Inselspital, Bern University Hospital, Bern, Switzerland
  • Vogt, Bruno, Inselspital, Bern University Hospital, Bern, Switzerland
  • Fuster, Daniel G., Inselspital, Bern University Hospital, Bern, Switzerland
Background

Previous HRQoL assessments in ADPKD patients yielded conflicting results. In addition, the impact of Tolvaptan treatment on HRQoL outcomes in ADPKD patients is currently unknown.

Methods

The Bern ADPKD registry is an observational cohort study initiated in 2015. Inclusion criteria are age ≥ 18y, clinical diagnosis of ADPKD, informed consent. The main exclusion criterion is need for renal replacement therapy. We assessed HRQoL of ADPKD patients with the standardized Short Form-36 (SF-36) as part of the KDQOL-SF™ 1.3 questionnaire in yearly intervals. The SF-36 consists of 8 multi-item subscales (physical functioning, role-physical, bodily pain, general health, energy/vitality, social functioning, role-emotional, mental health), and two summary scores: Physical Component Summary (PCS) and Mental Component Summary (MCS). We transformed raw scores into T-scores (mean=50, SD=10, range 0-100) using contemporaneous Swiss general population norms. Higher scores indicate better HRQoL.

Results

Between October 2015 and May 2019, 121 ADPKD patients were recruited and Tolvaptan treatment has been initiated in 38 patients. In six patients (16%) treatment had to be discontinued within the first three months of treatment due to aquaretic side effects (n=4, 11%) or due to elevated liver enzymes (n=2; 5%), and in eight patients (21%) a dose reduction was necessary.
We included 93 patients (28 with and 65 without Tolvaptan treatment) for which baseline and 1-year follow-up data were available. Thirty-nine patients were male (42%), median age was 45.7 y, median eGFR 70.4 ml/min and median total kidney volume 1197 ml. HRQoL at baseline was similar to the general population with a PCS of 48.6 (95% CI 46.2 – 51.0) and a MCS of 49.6 (95% CI 47.5 – 51.7). Patients with future Tolvaptan treatment had higher PCS scores than patients without future Tolvaptan treatment (52.8 versus 46.8; p<0.05). Individual subscales were not different. At 1 year follow-up HRQoL subscales, MCS and PCS scores were similar to baseline scores in both patients who received Tolvaptan and those who did not.

Conclusion

HRQoL in Swiss ADPDK patients is comparable to HRQoL in the general Swiss population. Furthermore, our data reveal that, beyond an initial adaptation to increased aquaresis, long-term treatment with Tolvaptan does not negatively affect HRQoL of ADPKD patients.

Funding

  • Commercial Support