Effects of metformin in adolescents with polycystic ovary syndrome undertaking lifestyle therapy: a pilot randomized double-blind study
Open accessAbstract
Our small study does not support the addition of metformin to the lifestyle of adolescents. Although there are favorable trends toward hyperandrogenism with metformin, these must be balanced against the increased rate of gastrointestinal side effects. However, other treatments were associated with an improved quality of life.
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Metformin may be considered when hyperinsulinemia is a dominant feature but is not a weight-loss medication per se.
“It may be considered when hyperinsulinemia is a dominant feature but is not a weight-loss medication per se [116].”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [116]
The paper reports no change in weight or waist circumference after 24 weeks and concludes that its study does not support adding metformin to lifestyle therapy in adolescents with PCOS (both passages labeled Abstract; page not supplied). This supports caution about treating metformin as a weight-loss medication in this setting, but the paper does not say it should be considered when hyperinsulinemia is a dominant feature. (LLM, conf=0.83)
Experimental — accuracy not yet measuredMetformin is used off-label in adolescents with insulin resistance or PCOS, with modest weight effects.
“Metformin is used off-label in adolescents with insulin resistance or PCOS, with modest weight effects.”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [116]
The study reports giving metformin to adolescents with PCOS and finding no significant change in weight; the discussion describes minimal weight gain in its participants. Our copy does not establish that this use was off-label or that the study treated adolescents for insulin resistance. The weight finding supports a limited effect in this small PCOS trial, not a general claim about adolescents. (LLM, conf=0.84)
Experimental — accuracy not yet measuredA meta-analysis of metformin use with and without lifestyle changes in PCOS (including two RCTs in adolescents) showed beneficial effects on BMI and menstrual cycles.
“A meta-analysis of metformin use with and without lifestyle changes in PCOS (including two RCTs in adolescents [164, 178]) showed beneficial effects on BMI and menstrual cycles [164, 178, 179].”
From Polycystic Ovary Syndrome: Pathophysiology, Presentation, and Treatment With Emphasis on Adolescent Girls (2019) · cites this paper as [178]
Another RCT by Ladson et al. on 22 adolescents found no significant differences in BMI between metformin plus lifestyle and placebo plus lifestyle.
“Another RCT by Ladson et al. 36 on 22 adolescents, comparing BMI changes among participants with metformin and lifestyle to those with placebo and lifestyle found no significant differences in BMI (Table SC).”
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“In addition, the addition of metformin has been associated with increased weight loss when combined with lifestyle interventions (6).”
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- Authors
- Ladson, G., Dodson, W. C., Sweet, S. D., Archibong, A. E., Kunselman, A. R., Demers, L. M., Lee, P. A., Williams, N. I., Coney, P., & Legro, R. S.
- Affiliations
- Meharry Medical College (US)
- Pennsylvania State University (US)
- Virginia Commonwealth University (US)
- Corresponding author
- Legro, Richard S.
- Venue
- Fertility and Sterility
- Publisher
- Elsevier BV
- Published
- 2011-06
- Volume
- 95
- Issue
- 8
- Pages
- 2595-2598.e6
- ISSN
- 0015-0282 (ISSN-L)
- PMC
- PMC3783023
- OpenAlex
- W2074835877
- Cited by
- 56
- References
- 27
- Citations by year
- 2026: 2 · 2025: 4 · 2024: 2 · 2023: 4 · 2022: 3 · 2021: 5 · 2020: 7 · 2019: 5 · 2018: 3 · 2017: 2 · 2016: 3 · 2015: 4 · 2014: 7 · 2013: 2 · 2012: 2
- FWCI
- 4.29
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- www.elsevier.com/tdm/userlicense/1.0/ (tdm)
- www.elsevier.com/legal/tdmrep-license (tdm)
- www.elsevier.com/open-access/userlicense/1.0/ (vor, 4013 d)
- doi.org/10.15223/policy-017 (stm-asf)
- doi.org/10.15223/policy-037 (stm-asf)
- doi.org/10.15223/policy-012 (stm-asf)
- doi.org/10.15223/policy-029 (stm-asf)
- doi.org/10.15223/policy-004 (stm-asf)
- Locations
- Fertility and Sterility (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Pennsylvania State University
- Topics
- Health Sciences › Medicine › Reproductive Medicine › Ovarian function and disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and Kidney Cyst Diseases
- Health Sciences › Medicine › Reproductive Medicine › Endometriosis Research and Treatment
- Keywords
- metformin, polycystic ovary syndrome, adolescents, lifestyle therapy, hyperandrogenism, gastrointestinal adverse effects, quality of life, insulin sensitizers
- MeSH terms
- Adolescent, Combined Modality Therapy, Double-Blind Method, Exercise Therapy, Female, Humans, Metformin, Pilot Projects, Polycystic Ovary Syndrome, Quality of Life, Regression Analysis, Risk Factors, Testosterone, Time Factors, United States, Biomarkers, Treatment Outcome, Risk Assessment, Caloric Restriction, Risk Reduction Behavior
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