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The following is a summary of “Long-term effect of tolvaptan treatment on kidney function and volume in patients with ADPKD,” published in the March 2025 issue of Nephrology Dialysis Transplantation by Geertsema et al. 

Tolvaptan is the only therapy that slows disease progression in autosomal dominant polycystic kidney disease (ADPKD). Real-life long-term data are limited by short follow-up, small cohorts, or missing controls. 

Researchers conducted a retrospective study on the long-term effect of tolvaptan on kidney function and growth in real-life patients and controls. They also analyzed factors influencing treatment efficacy. 

They pooled data from the DIPAK and OBSERVA cohorts. eGFR was measured yearly, and total kidney volume (TKV) every 3 years. Treatment effects from baseline to 6 weeks were analyzed as ‘acute slope’ and after as ‘chronic slope.’ Controls included untreated patients, assessing endpoint changes before and during theoretical treatment based on average tolvaptan initiation time. 

The results showed that 615 patients (48 ± 12 years, 58.2% female) were analyzed, with 105 (17.1%) receiving tolvaptan for an average of 6.1 ± 4.7 years (range: 0.8–15.9). After matching, 92 patients per group remained. Tolvaptan reduced eGFR decline by 14.0% (-4.36 to -3.75 mL/min/1.73m² /year, P = 0.03), while controls showed a 1.0% decrease (-4.16 to -4.12 mL/min/1.73 m² /year, P = 0.9). Long-term TKV growth remained unchanged (5.05 to 5.59%/year, P = 0.6). Higher osmolar intake correlated with a greater treatment effect. 

Investigators found that tolvaptan attenuated long-term kidney function decline but did not significantly affect kidney growth. 

Source: academic.oup.com/ndt/advance-article/doi/10.1093/ndt/gfaf048/8058887