Cabozantinib Effective as Salvage Therapy in Thyroid Cancer

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Significant clinical benefit was found in some patients treated with cabozantinib in the salvage setting.
Significant clinical benefit was found in some patients treated with cabozantinib in the salvage setting.

Patients with radioiodine (RAI)-refractory differentiated thyroid cancer (DTC) may experience significant clinical benefit if treated with cabozantinib in the salvage setting, according to a study published in the Journal of Clinical Oncology.1

There is a lack of secondary therapies available for patients with thyroid cancer, and available therapies in second/third-line or salvage setting possess poor toxicity profiles or are inadequately able to sustain response to treatment. A previous phase 1 study demonstrated that cabozantinib may have potential for patients with DTC who have failed previous vascular endothelial growth factor receptor (VEGFR)-targeted treatment.

The researchers for this phase 2 study ( Identifier: NCT01811212) administered oral cabozantinib 60 mg once daily to 25 eligible patients with DTC. Twenty-one patients had failed one line of VEGFR-targeted therapy, and 4 patients had failed two lines. 

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Ten (40%) of the 25 study patients had a partial response (PR), and 13 (52%) patients had stable disease (SD). The median time to achieve PR was 2 months, and median duration of PR was 11.3 months (95% CI, 10.3-not evaluable).

The median duration of follow-up was 22.8 months (95% CI, 21.2-30.2), at which point the median progression-free survival was 12.7 months (95% CI, 10.9-34.7). The median overall survival (OS) was 34.7 months (95% CI, 18.3-not reached).

RELATED: USPSTF Recommends Against Thyroid Cancer Screening

One patient died due to treatment-related causes. The most frequently reported adverse events were weight loss, fatigue, diarrhea, hypertension, and palmar-plantar erythrodysesthesia.


  1. Cabanillas ME, de Souza JA, Geyer S, et al. Cabozantinib as salvage therapy for patients with tyrosine kinase inhibitor-refractory differentiated thyroid cancer: results of a multicenter phase II international thyroid oncology group trial. J Clin Oncol. 2017 Aug 17. doi: 10.1200/JCO.2017.73.0226. [Epub ahead of print]

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