Tolvaptan may slow eGFR decline in patients with autosomal dominant PKD
Key takeaways:
- Patients on tolvaptan had an eGFR decline of 1.40 mL/min/1.73 m² per year.
- Annual rate of eGFR decline in patients on tolvaptan was slower than in historical controls.
Routine use of tolvaptan in clinical practice may slow eGFR decline for patients with autosomal dominant polycystic kidney disease compared with historical controls, data show.
“Efficacy of tolvaptan has been demonstrated in controlled clinical trials with highly motivated patients and regular follow-up at PKD centers of excellence,” Ronald D. Perrone, MD, FASN, professor of medicine at the Tufts University School of Medicine in the division of nephrology, told Healio. “The finding of similar efficacy in clinical practice, involving multiple clinicians distributed over varied geographic locations, indicates that the benefit observed in trials translates to clinical use.”


Researchers performed a chart review for 149 U.S. patients with autosomal dominant PKD (ADPKD) treated with tolvaptan for at least 2 years. Data for these patients were matched to a historical control cohort who received standard care without tolvaptan, including various antihypertensive regimens.
Controls were in Mayo imaging classes 1C through 1E and were at high risk for rapid progression.
In an online survey, 57 U.S.-based nephrologists provided data for patients treated with tolvaptan. Control data for patients with ADPKD not treated with tolvaptan were sourced from clinical studies.
Nephrologists provided kidney function measurements for up to 4 years of follow-up. Main outcome was annual eGFR change.
Researchers found the annual rate of eGFR decline in patients on tolvaptan was significantly slower than that of historical controls. In an initial analysis set, patients on tolvaptan had an eGFR decline of 1.40 mL/min/1.73 m² per year (95% CI, 0.05-2.74). A second analysis set confirmed this finding, showing an eGFR decline of 1.18 mL/min/1.73 m² per year (95% CI, 0.22 to 2.58), though this was not statistically significant.
“The finding of similar results in clinical trials and clinical practice confirms the efficacy of tolvaptan to slow GFR decline in ADPKD patients at risk of rapid progression,” Perrone said. “Results also confirm that use of tolvaptan in clinical practice is feasible and effective.”