Metformin as a Disease-Modifying Agent in Autosomal Dominant Polycystic Kidney Disease: A Systematic Review of Preclinical and Clinical Evidence.

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is a common inherited kidney disorder marked by cyst growth and progressive renal failure. This systematic review aims to summarize current preclinical and clinical evidence on the potential role of metformin in ADPKD, focusing on its effects on glucose metabolism, kidney function, inflammation, and survival. A comprehensive search was conducted in PubMed and Google Scholar up to June 2025, following PRISMA guidelines. Forty-two articles met the inclusion criteria and were analyzed. Included studies examined metformin use in ADPKD patients or models and reported outcomes such as renal function, cyst growth, metabolic markers, and mortality. In preclinical studies, it reduced cyst formation, improved kidney structure, and decreased inflammation. Clinical studies confirmed its safety and suggested benefits in slowing kidney function decline, especially in early-stage ADPKD. Metformin may be a promising supportive therapy in ADPKD due to its metabolic and anti-inflammatory effects.