Effect of tolvaptan on the prognosis of patients with rapidly progressive autosomal dominant polycystic kidney disease: a prospective open-label study

Introduction Although tolvaptan is internationally recognized for its therapeutic benefits in autosomal dominant polycystic kidney disease (ADPKD) and has been referenced in the 2020 Chinese Clinical Practice Guidelines, it has yet to be approved in China due to limited large-scale local evidence. Methods A two-year, prospective, single-center, open-label trial was conducted to assess the efficacy and safety of tolvaptan in patients with rapidly progressive ADPKD. The primary endpoint was the change in total kidney volume (TKV), while secondary endpoints included changes in eGFR and evaluation of safety and tolerability. Results Eighty-nine patients were enrolled 80% had a family history of ADPKD, 40% had polycystic liver disease and 65.6% were diagnosed with hypertension. A proportion of patients also presented with renal insufficiency and vascular complications. Baseline laboratory tests showed a decreased median red blood cell count and elevated concentrations of urinary albumin, urea, creatinine and uric acid. The most commonly used daily doses of tolvaptan were 60 mg (31.1%) and 45 mg (26.7%). After treatment, the mean TKV change rate for the left kidney was −70.2 ± 206.07 mL at 6 months and −47.84 ± 132.58 mL at 12 months; for the right kidney, the respective changes were −42.69 ± 209.40 mL and −8.57 ± 286.86 mL. eGFR remained stable, with no major hepatic/renal toxicity. Conclusion Tolvaptan effectively slowed kidney volume growth and was well tolerated in Chinese patients with rapidly progressive ADPKD.