What is the role of pharmacologic therapy in the treatment of thyroid cancer?

Updated: May 14, 2020
  • Author: Pramod K Sharma, MD; Chief Editor: Arlen D Meyers, MD, MBA  more...
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Sorafenib (Nexavar) was approved in November 2013 for differentiated thyroid cancer (DTC) that is refractory to radioactive iodine treatment. In a study of 417 patients with progressive radioiodine-refractory DTC, treatment with sorafenib, an orally active inhibitor of VEGFR1-3 and Raf kinases, significantly improved progression-free survival (10.8 months) compared with placebo (5.8 months). [20, 21, 22] Tumor histology was 57% papillary, 25% follicular, and 10% poorly differentiated. The majority of the patients (96%) had metastatic disease, of which 71% of the target lesions were in the lung, 40% in lymph nodes, and 14% in bone.

At the time of the report, median overall survival had not yet been reached in either study arm, and 70% of placebo patients had started open-label sorafenib. [20, 21] Thus, all reported responses were partial: 12.2% in the sorafenib group vs 0.5% in the placebo group. The rate of stable disease for 6 months or longer was 42% in the sorafenib group and 33% in the placebo group.

A second oral VEGF inhibitor for refractory DTC was approved, in February 2015. Approval for lenvatinib (Lenvima) was based on the SELECT phase-3 trial. Compared with patients on placebo, those taking lenvatinib showed significant improvements in progression-free survival (median period of 18.3 months, compared with 3.6 months with placebo). The response rate among patients with iodine-131-refractory thyroid cancer was also significantly better with lenvatinib than with placebo. [23, 24]

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