Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2023 Mar;55(3):631-640.
doi: 10.1007/s11255-022-03353-8. Epub 2022 Sep 7.

Efficacy and safety of tolvaptan versus placebo in the treatment of patients with autosomal dominant polycystic kidney disease: a meta-analysis

Jingkui Lu  1   2 , Wei Xu  3   4 , Lifeng Gong #  1   2 , Min Xu #  1   2 , Weigang Tang  1   2 , Wei Jiang  1   2 , Fengyan Xie  1   2 , Liping Ding  1   2 , Xiaoli Qian  1   2
Affiliations
Review

Efficacy and safety of tolvaptan versus placebo in the treatment of patients with autosomal dominant polycystic kidney disease: a meta-analysis

Jingkui Lu et al. Int Urol Nephrol. 2023 Mar.

Abstract

Objective: The objective of this meta-analysis was to compare the efficacy and drug safety of tolvaptan with placebo for autosomal dominant polycystic kidney disease (ADPKD).

Methods: The PubMed, Embase, and Cochrane Library databases were searched from inception to September 10, 2021. Eligible studies comparing tolvaptan and placebo in the treatment of patients with ADPKD were included. Data were analysed using Review Manager Version 5.3.

Results: Thirteen studies involving 3575 patients were included in the meta-analysis. Compared with placebo, tolvaptan had a better effect on delaying eGFR decline (MD 1.27, 95% CI 1.24-1.29, P < 0.01) and TKV increase (MD - 3.01, 95% CI - 3.55 to - 2.47, P < 0.01) in ADPKD treatment. Additionally, tolvaptan reduced the incidence of complications such as renal pain (OR 0.71, 95% CI 0.58-0.87, P < 0.01), urinary tract infection (OR 0.69, 95% CI 0.54-0.89, P < 0.01), haematuria (OR 0.68, 95% CI 0.51-0.89, P < 0.01), and hypertension (OR 0.66, 95% CI 0.52-0.82, P < 0.01). However, tolvaptan was associated with a higher incidence rate of adverse events such as thirst (OR 8.48 95% CI 4.53-15.87, P < 0.01), polyuria (OR 4.71, 95% CI 2.17-10.24, P < 0.01), and hepatic injury (OR 4.56, 95% CI 2.51-8.29, P < 0.01).

Conclusion: Tolvaptan can delay eGFR decline and TKV increase and reduce complications such as renal pain, urinary tract infection, haematuria, and hypertension in the treatment of ADPKD. However, tolvaptan increases the adverse effects of thirst, polyuria and hepatic injury.

Keywords: Autosomal dominant polycystic kidney disease; Meta-analysis; Placebo; Tolvaptan.

PubMed Disclaimer

Conflict of interest statement

The authors declare that they have no conflicts of interest.

Figures

Fig. 1
Fig. 1
Flow diagram of the literature search
Fig. 2
Fig. 2
Forest plot of eGFR slope (ml/min per 1.73 m2 per year) between tolvaptan and placebo
Fig. 3
Fig. 3
Forest plot of TKV increase rate (%/year) between tolvaptan and placebo
Fig. 4
Fig. 4
Forest plot of renal pain between tolvaptan and placebo
Fig. 5
Fig. 5
Forest plot of urinary tract infection between tolvaptan and placebo
Fig. 6
Fig. 6
Forest plot of haematuria between tolvaptan and placebo
Fig. 7
Fig. 7
Forest plot of hypertension between tolvaptan and placebo
Fig. 8
Fig. 8
Forest plot of thirst between tolvaptan and placebo
Fig. 9
Fig. 9
Forest plot of polyuria between tolvaptan and placebo
Fig. 10
Fig. 10
Forest plot of hepatic injury between tolvaptan and placebo
See this image and copyright information in PMC

References

    1. Bergmann C, Guay-Woodford LM, Harris PC, et al. Polycystic kidney disease. Nat Rev Dis Primers. 2018;4(1):50. doi: 10.1038/s41572-018-0047-y. - DOI - PMC - PubMed
    1. Sommerer C, Zeier M. Clinical manifestation and management of ADPKD in western countries. Kidney Dis (Basel) 2016;2(3):120–127. doi: 10.1159/000449394. - DOI - PMC - PubMed
    1. Ong AC, Devuyst O, Knebelmann B, Walz G, et al. ERA-EDTA Working Group for Inherited Kidney Diseases: autosomal dominant polycystic kidney disease: The changing face of clinical management. Lancet. 2015;385:1993–2002. doi: 10.1016/S0140-6736(15)60907-2. - DOI - PubMed
    1. Torres VE, Harris PC, Pirson Y, et al. Autosomal dominant polycystic kidney disease. Lancet. 2007;369:1287–1301. doi: 10.1016/S0140-6736(07)60601-1. - DOI - PubMed
    1. Grantham JJ. Autosomal dominant polycystic kidney disease. N Engl J Med. 2008;359:1477–1485. doi: 10.1056/NEJMcp0804458. - DOI - PubMed

MeSH terms