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Kidney Week

Abstract: PO1536

Impact of Long-Term Tolvaptan Treatment for Autosomal Dominant Polycystic Kidney Disease: A Single-Centre Retrospective Japanese Cohort Study

Session Information

Category: Genetic Diseases of the Kidneys

  • 1001 Genetic Diseases of the Kidneys: Cystic

Authors

  • Yamamoto, Junya, Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Hokkaido, Japan
  • Nishio, Saori, Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Hokkaido, Japan
  • Nakazawa, Daigo, Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Hokkaido, Japan
  • Atsumi, Tatsuya, Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Hokkaido, Japan
Background

Several clinical trials have revealed the efficacy of the tolvaptan for autosomal dominant polycystic kidney disease (ADPKD) in recent years. Our objective is to verify the impact of tolvaptan in our Japanese ADPKD cohort.

Methods

We retrospectively investigated the efficacy of tolvaptan for ADPKD patients who initiated tolvaptan from June 2014 to March 2020 in Hokkaido University Hospital. Patients treated with tolvaptan for more than 1 year were included for analyses. Patients never treated by tolvaptan were set as control. Patients in CKD stage 5 or 5D at baseline or postkidney transplantation were excluded from analyses. We stratified patients by Mayo classification (Class 1A-1E). Analyses included the comparison of the annual changes of eGFR(ΔeGFR (mL/min/1.73m2/year)) and total kidney volume (ΔTKV (%/year)) between pre and post-treatment, and ΔeGFR or ΔTKV between tolvaptan-treated and control.

Results

109 tolvaptan-treated and 139 control patients were included. 24 patients in each group were excluded. About 40% of tolvaptan-treated patients belonged to advanced CKD stage (CKD3b-4). Duration of tolvaptan treatment was 3.3±1.3 years. eGFR of tolvaptan group were lower and htTKV of tolvaptan group were higher compared to those of control group at baseline (eGFR: 53.7±22.8 vs 65.7±30.0, p=0.16. htTKV: 1193.9±649.6 vs 829.5±799.5 mL/m, p<0.0001). There was no significant difference in ΔeGFR between (tolvaptan -2.95±2.86 vs control -3.09±3.31, p=0.33), however in tolvaptan group ΔeGFR improved compared to pre-treatment (-2.95±2.86 vs -4.33±5.72, p=0.027) and this improvement lasted at least for 36 months in 50 patients. ΔTKV of tolvaptan group was lower than that of control (2.19, 95%CI(0.33-4.05) vs 5.10(4.00-6.19), p<0.01) and this trend was also found in Mayo class 1B-1D. ΔTKV also decreased compared to pre-treatment (2.19(0.33-4.06) vs 4.67(2.57-6.76), p<0.01).

Conclusion

Tolvaptan dramatically reduced ΔTKV, while there was no beneficial effect for ΔeGFR compared to control. However tolvaptan improved ΔeGFR compared to that of pre-treatment. The discrepancy between our results and previous reports might arise from the fact that our cohort mainly comprised advanced CKD stage patients or limited sample size. Further long-term observation is required to validate the effect of tolvaptan.