Background: Tolvaptan is the first drug approved by the FDA for rapidly progressive polycystic kidney disease (PKD). However, there are no studies reporting real-world clinical practice using tolvaptan to treat Chinese patients with autosomal-dominant polycystic kidney disease (ADPKD). Methods: This retrospective multicentre cohort study analyzed ADPKD patients diagnosed by genetic or imaging tests, comparing those treated with tolvaptan(15 mg, twice daily) to unmedicated patients. Clinical course, estimated glomerular filtration rate (eGFR), total kidney volume (TKV), SF-36 score, and adverse events (AEs) were recorded for 83 patients, with 40 in the treatment group and 43 in the control group. Results: Compared with the control group, the tolvaptan treatment significantly delayed the increase in height-adjusted total kidney volume growth rate (htTKV%) in the 1st and 2nd years (1 year: difference:-5.58, 95% confidence interval (CI): -8.39 to -2.78, p < 0.001; 2 year: difference: -9.75, 95% CI: -13.1 to -6.31), p < 0.001). Compared with the control group, the effect of tolvaptan on eGFR was more pronounced in the 2nd year of treatment (2 year: difference: 6.79, 95% CI: 5.10-8.48, p < 0.001). Tolvaptan attenuated the AEs, such as fatigue, back pain, and anxiety, to some extent, while the incidence of water-related AEs, such as thirst, polyuria, and urinary frequency, was significantly higher than that in the control group (p < 0.001, p = 0.006, p = 0.009, respectively). According to the Short-Form 36 (SF-36) health survey, the physical component summary (PCS) score was significantly improved in the treatment group (p < 0.001). Conclusion: This real-world analysis showed that tolvaptan appears to be effective in delaying the clinical course of ADPKD patients in China, extending the results of previous clinical trials in other populations.
Li et al. (Tue,) studied this question.