Quality of Life in Autosomal Dominant Polycystic Kidney Disease Patients Treated With Tolvaptan

Abstract

Rationale & Objective

The impact of tolvaptan on health-related quality-of-life (HRQoL) in patients with autosomal dominant polycystic kidney disease (ADPKD) is unknown. To address this knowledge gap, we studied patient-reported HRQoL in patients enrolled in the Bern ADPKD registry.

Study Design

Prospective cohort study.

Settings & Participants

Inclusion criteria were age 18 years or older, clinical diagnosis of ADPKD, and informed consent. The main exclusion criterion was need for kidney replacement therapy.

Outcome

HRQoL was assessed using the standardized Kidney Disease Quality of Life Short Form (KDQOL-SF) questionnaire at start of the study (baseline) and after 1 year (follow-up). The KDQOL-SF has 2 parts: a generic 36-Item Health Survey instrument with 8 subscores and 2 summary scores and a kidney disease–specific instrument to assess health concerns. Higher scores indicate better HRQoL. The influence of tolvaptan treatment on HRQoL and kidney-specific health concerns was analyzed using analysis of covariance, adjusting for HRQoL and health concerns before the start of the study, sex, and age.

Results

In 38 of 121 registry patients, tolvaptan treatment was initiated. Within the first 3 months, treatment had to be discontinued in 6 (16%) patients due to aquaretic side effects (n = 4; 11%) or elevated liver enzyme levels (n = 2; 5%), and a dose reduction was necessary in 8 (21%) patients. We included 98 patients (30 with and 68 without tolvaptan treatment) in the analysis for which baseline and 1-year follow-up data were available. At follow-up, and after adjusting for baseline scores, sex, and age, HRQoL and kidney-specific health concerns were not influenced by tolvaptan treatment, except for patient satisfaction, which was increased.

Limitations

Observational study design, monocentric study at tertiary referral hospital, almost exclusively white study population, grant support by Otsuka Pharmaceuticals.

Conclusions

Our results indicate that tolvaptan does not significantly affect HRQoL in patients with ADPKD who tolerate treatment beyond the first 3 months of therapy.

Graphical abstract