Thyroid disease

Drug Repurposing for Reducing the Size of Benign Thyroid Nodules: A Systematic Review

J Clin Endocrinol MetabPublished Systematic review

Authors' conclusion

Metformin shows the most consistent potential for noninvasive reduction of BTN size, particularly in patients with metabolic comorbidities. However, current evidence is limited and heterogeneous. Further large-scale studies are needed to confirm efficacy and guide clinical use.

Study context

Population
Adults with benign thyroid nodules (BTNs); preclinical and clinical studies of repurposed drugs20 studies
Comparison
Repurposed pharmacologic agents for reducing BTN size
Outcome
Reduction in BTN size
Methodological detailsnarrative synthesis, Data extracted independently in duplicate, MEDLINE, EMBASE, Cochrane and Scopus searched from inception to December 18, 2024, 1499 records screened; 20 included: 6 randomized controlled trials (RCTs), 6 prospective cohorts, 3 retrospective observational, 3 case reports, 1 cross-sectional, 1 preclinical
  • Narrative synthesis
  • Data extracted independently in duplicate
  • MEDLINE, EMBASE, Cochrane and Scopus searched from inception to December 18, 2024
  • 1499 records screened; 20 included: 6 RCTs, 6 prospective cohorts, 3 retrospective observational, 3 case reports, 1 cross-sectional, 1 preclinical

Pooled result

As reported in the abstract

BTN size
Metformin (most evaluated agent): significant reductions in dominant nodule volume
5 of 7 studies (including 2 RCTs)
Metformin: non-significant reduction in maximum nodule diameter
1 prospective study
Metformin: no changes in nodule size
1 RCT
Statins: mixed results
4 studies
Somatostatin analogues: mixed results
4 studies

Original publication

Drug Repurposing for Reducing the Size of Benign Thyroid Nodules: A Systematic Review. J Clin Endocrinol Metab. Published 12 November 2025. doi:10.1210/clinem/dgaf616

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