The Journal of clinical endocrinology and metabolism · 2026
Drug Repurposing for Reducing the Size of Benign Thyroid Nodules: A Systematic Review.
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Soto Jacome Cristian, Arce-Camposano Andrea, Toro-Tobon David, Al Zahidy Misk, Rivadeneira Francisco, Fierro Francisco E, Montero Marcelo, Ponce Oscar, Singh Ospina Naykky, Brito Juan P
Аннотация
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 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.
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 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.