Metformin Active Surveillance Trial in Low-Risk Prostate Cancer

N Neil E. Fleshner (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) R Rui M. Bernardino (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) J Jonathan Izawa (University of Western Ontario, London, ON, Canada) D Darrel Drachenberg (Manitoba Prostate Cancer, University of Manitoba, Winnipeg, MB, Canada) J Jeff W. Saranchuk (CancerCare Manitoba, Winnipeg, MB, Canada) A Adrian Fairey (Northern Alberta Urology Clinic, Edmonton, AB, Canada) S Simon Tanguay (McGill University Health Centre, Montréal, QC, Canada) M Michael Leveridge (Queen's University, Kingston, ON, Canada) F Fred Saad (Centre Hospitalier de l’Université de Montréal, University of Montreal, Montreal) R Rodney H. Breau (University of Ottawa, Ottawa) B Bobby Shayegan L Laurence Klotz (Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada) K Karen Hersey (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) S Sunakshi Chowdhary (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) K Karen Chadwick (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) H Heidi Wagner (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) T Tiiu Sildva (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) S Shabbir M.H. Alibhai N Naghmeh Rastegar (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) M Miran Kenk (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) R Rosette Veloso (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) J Jessica G. Cockburn (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) J Joan Sweet (Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada) C Clare O'Connell (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) L Linda Kapusta (Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada) A Aingeshaan Kubendran (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) T Tabassom Azizi (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) D Doron Berlin (Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) R Robert J. Hamilton (Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) K Katherine Lajkosz (Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada) T Theodorus van der Kwast (Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada) R Ricardo A. Rendon (Department of Urology, Dalhousie University, Halifax, NS, Canada) P Patrick O. Richard A Anthony M. Joshua (Immunology Division, Garvan Institute of Medical Research)

Abstract

PURPOSE Active surveillance (AS) is a standard management strategy for low-risk prostate cancer (PCa), but a significant proportion of patients ultimately experience disease progression. Metformin, a commonly prescribed antidiabetic agent, has demonstrated antitumor activity in preclinical studies and observational data, prompting investigation into its potential to delay PCa progression. PATIENTS AND METHODS The Metformin Active Surveillance Trial (MAST) was a multicenter, randomized, double-blind, placebo-controlled phase III trial evaluating the efficacy of metformin in men with low-risk, localized PCa managed with AS. Eligible participants were randomly assigned 1:1 to receive either metformin (850 mg twice daily) or placebo and were followed for up to 36 months. The primary end point was time to progression, defined as therapeutic and/or pathologic progression. Progression-free survival (PFS) was assessed using Kaplan-Meier analysis and Cox proportional hazards models. RESULTS A total of 408 patients were randomly assigned (205 metformin, 203 placebo). After a median follow-up of 36 months, 144 participants experienced progression (70 metformin, 74 placebo), with no significant difference in PFS (hazard ratio [HR], 1.09 [95% CI, 0.79 to 1.52]; P = .59). Negative biopsy rates at 36 months were 41.0% (metformin) versus 31.1% (placebo; P = .181). In prespecified subgroup analysis, metformin was associated with increased pathologic progression among obese patients (BMI ≥ 30; HR, 2.36 [95% CI, 1.21 to 4.59]; P = .0092). CONCLUSION Metformin did not reduce progression in men with low-risk PCa on AS. The observed adverse effect in obese patients merits further investigation.

Article Details

Volume / Issue Vol. 43, Issue 34
Published December 01, 2025
Pages 3662-3671
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (34)

N

Neil E. Fleshner

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

R

Rui M. Bernardino

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

J

Jonathan Izawa

University of Western Ontario, London, ON, Canada

D

Darrel Drachenberg

Manitoba Prostate Cancer, University of Manitoba, Winnipeg, MB, Canada

J

Jeff W. Saranchuk

CancerCare Manitoba, Winnipeg, MB, Canada

A

Adrian Fairey

Northern Alberta Urology Clinic, Edmonton, AB, Canada

S

Simon Tanguay

McGill University Health Centre, Montréal, QC, Canada

M

Michael Leveridge

Queen's University, Kingston, ON, Canada

F

Fred Saad

Centre Hospitalier de l’Université de Montréal, University of Montreal, Montreal

R

Rodney H. Breau

University of Ottawa, Ottawa

B

Bobby Shayegan

L

Laurence Klotz

Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada

K

Karen Hersey

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

S

Sunakshi Chowdhary

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

K

Karen Chadwick

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

H

Heidi Wagner

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

T

Tiiu Sildva

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

S

Shabbir M.H. Alibhai

N

Naghmeh Rastegar

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

M

Miran Kenk

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

R

Rosette Veloso

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

J

Jessica G. Cockburn

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

J

Joan Sweet

Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada

C

Clare O'Connell

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

L

Linda Kapusta

Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada

A

Aingeshaan Kubendran

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

T

Tabassom Azizi

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

D

Doron Berlin

Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

R

Robert J. Hamilton

Division of Urologic Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

K

Katherine Lajkosz

Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada

T

Theodorus van der Kwast

Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada

R

Ricardo A. Rendon

Department of Urology, Dalhousie University, Halifax, NS, Canada

P

Patrick O. Richard

A

Anthony M. Joshua

Immunology Division, Garvan Institute of Medical Research