Endocrine oncology

Efficacy and Safety of Targeted Therapy for Radioiodine-Refractory Differentiated Thyroid Cancer

J Clin Endocrinol MetabPublished Systematic review and meta-analysis of randomised trials and single-arm studies

An erratum to this article was published (J Clin Endocrinol Metab, 16 Sep 2025 ↗)

Authors' conclusion

The currently used targeted drug therapies for RR-DTC can significantly improve clinical outcomes, and the new drug apatinib demonstrates promise for potentially superior performance.

Study context

Population
Patients with radioiodine-refractory DTC (radioiodine-refractory differentiated thyroid cancer (RR-DTC))
Participants
3,270 patients8 randomized controlled trials (RCTs)17 single-arm studies
Comparison
Targeted drug therapy vs control in RR-DTC
Outcome
Progression-free survival (PFS) and overall survival (OS)

Pooled result

As reported in the abstract

Estimate (95% CI)P value
Targeted therapy
HR (PFS) 0.35(0.23–0.53)
P < 0.00001
Targeted therapy
HR (OS) 0.53(0.32–0.86)
P < 0.00001

Further pooled results

Objective response rate (ORR) and disease control rate (DCR)

EstimateP value
Targeted therapyRR (ORR) 27.63 (95% CI 12.39–61.61)P < 0.00001
Targeted therapyRR (DCR) 1.66 (95% CI 1.48–1.86)P < 0.00001

Apatinib subgroup: ORR and DCR (effect size)

Apatinib ORR effect size (ES)
0.66 (95% CI 0.49-0.83, P < .00001)
Apatinib DCR ES
0.95 (95% CI 0.91-1.00, P < .00001)
  • Apatinib had the best effect on ORR and DCR

Safety

Common adverse events: hypertension, diarrhea, proteinuria, fatigue

Original publication

Efficacy and Safety of Targeted Therapy for Radioiodine-Refractory Differentiated Thyroid Cancer. J Clin Endocrinol Metab. Published 18 September 2024. doi:10.1210/clinem/dgae617

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