A secondary analysis of concurrent use of metformin and tolvaptan in ADPKD tolvaptan trials

Stanley, I. Kitty, Palma, Anton M., Viecelli, Andrea K., Johnson, David W., Hawley, Carmel M., Staatz, Christine E., and Mallett, Andrew J. (2024) A secondary analysis of concurrent use of metformin and tolvaptan in ADPKD tolvaptan trials. Journal of Nephrology, 37 (5). pp. 1417-1419.

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Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the leading genetic cause of kidney disease [1]. Tolvaptan, a vasopressin V2-receptor antagonist, is the only disease-modifying therapy available with proven efficacy in slowing disease progression. The anti-diabetic medication, metformin, has been theorised to be of potential benefit in ADPKD. Metformin activates 5 AMP-activated protein kinase, which negatively regulates the cystic fibrosis transmembrane conductance regulator and the mammalian target of rapamycin, both of which are implicated in the growth of cysts in ADPKD [2]. Two phase II trials [3, 4] have found metformin to be safe and tolerable in patients with ADPKD, with a non-significant slowing of estimated glomerular filtration rate (eGFR) decline and total kidney volume expansion. A phase III trial is currently underway to further define metformin’s role in ADPKD [5]. Given the established role of tolvaptan and the potential role of metformin in the management of ADPKD, the safety and efficacy of combined therapy are also of interest.

Item ID: 87263
Item Type: Article (Short Note)
ISSN: 1724-6059
Copyright Information: This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/
Date Deposited: 12 Nov 2025 00:59
FoR Codes: 32 BIOMEDICAL AND CLINICAL SCIENCES > 3202 Clinical sciences > 320214 Nephrology and urology @ 100%
SEO Codes: 20 HEALTH > 2001 Clinical health > 200105 Treatment of human diseases and conditions @ 100%
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