Aims and Objective: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a
common chronic kidney disease that leads to End-Stage Renal Disease (ESRD). The key target of
this therapy is to prevent the progression of kidney failure. Tolvaptan could slow kidney cyst
growth and are proven highly effective. The aims of this analysis are to perform a systematic
review, estimate and evaluate the efficacy and safety of tolvaptan in ADPKD patients.
Materials and Methods: Randomized controlled trials of tolvaptan in ADPKD were identified in
PubMed, Ovid, Web of Science and the Cochrane Library electronic database. The changes
observed in kidney function, treatment efficiency and the incidence of adverse events between the
tolvaptan and placebo groups were compared. Data were analyzed by the RevMan software.
Results: Eight trials, including 7 double-blinded randomised controlled trials and 1 quasi RCT
involving 1,536 patients were extracted. Significant differences in the annual rate of change in the
total kidney volume TKV at any stages of CKD (MD = -3.32, 95%CI =-4.57,-2.07, I2 =70%) and
the glomerular filtration rate (MD = 1.4, 95%CI = 0.83,1.97, I2 =0%) were observed between the
tolvaptan group and the placebo group. Subgroup analysis of patients in different CKD stages also
showed the same conclusion. There was an increase in the urine osmolality, and 24-hour urine
volume in patients receiving tolvaptan. Tolvaptan reduced the rate of serious hypertension and
kidney pain events in ADPKD patients. At higher doses, it increased the rate of adverse events
(liver injuries, thirst, pollakiuria, and nocturia). There was no significant risk of bias in the
Conclusion: Tolvaptan has a beneficial effect on ADPKD, but is associated with an increase in
adverse events at high doses when compared with the placebo. Further RCTs on tolvaptan may be
required to support this conclusion.