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

Soto Jacome C, Arce-Camposano A, Toro-Tobon D, Al Zahidy M, Rivadeneira F, Fierro FE, Montero M, Ponce O et al.

The Journal of clinical endocrinology and metabolism, 2026

1 citation

Abstract

Context: Benign thyroid nodules (BTNs) are highly prevalent, and current treatment options are primarily invasive. There is a need for effective, noninvasive alternatives.

Objective: To systematically evaluate the efficacy of repurposed pharmacologic agents in reducing the size of BTNs.

Data sources: MEDLINE, EMBASE, Cochrane databases, and Scopus were searched from inception to December 18, 2024.

Study selection: We included preclinical and clinical studies evaluating repurposed drugs for BTNs in adults. Of 1499 records screened, 20 studies met inclusion criteria: 6 randomized controlled trials (RCTs), 6 prospective cohorts, 3 retrospective observational studies, 3 case reports, 1 cross-sectional study, and 1 preclinical study.

Data extraction: Data were extracted independently in duplicate using a standardized form.

Data synthesis: Narrative synthesis was conducted due to heterogeneity. Metformin was the most frequently evaluated agent (7 studies), with 5 studies (including 2 RCTs) reporting statistically significant reductions in dominant nodule volume. One prospective study demonstrated a reduction in the maximum nodule diameter, which was not statistically significant, and 1 RCT showed no changes in nodule size after intervention. Statins (4 studies) and somatostatin analogues (4 studies) showed mixed results. Myo-inositol with selenium, herbal formulations, and immune checkpoint inhibitors showed promise in single studies or case reports.

Conclusion: Metformin shows…

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Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid.

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