Treatment with sorafenib in patients with progressive metastatic medullary thyroid cancer (MTC) is well tolerated and resulted in disease control and durable clinical benefit in 75% of patients, a study published online ahead of print in the journal Thyroid has shown.1

Although treatment of advanced MTC has recently improved with the approval of vandetanib and cabozantinib, there is still a need to investigate alternative options and explore sequential therapies with more than 1 tyrosine kinase inhibitor (TKI). Therefore, researchers sought to evaluate the safety and efficacy of sorafenib as a treatment for advanced MTC.

For the retrospective study, researchers analyzed data from 12 patients with progressive metastatic MTC, of which 10 had sporadic and 2 had hereditary disease. All patients had received sorafenib 400 mg twice daily between December 2011 and January 2015.

Continue Reading

Results showed that 83.3% of patients showed stable disease at follow-up, 16.6% had progressive disease, and no patients had achieved a partial response. Researchers found that median progression-free survival was 9 months, but they note that 3 patients with extensive and rapidly progressive disease had died within 3 months of sorafenib therapy. When excluding these patients, the median progression-free survival was 12 months.

In regard to safety, adverse events were reported in 75% of patients, with the most frequent being skin toxicity, weight loss, and fatigue. Toxicity led to dose reductions in 5 patients and treatment discontinuation in 1 patient.

The findings suggest that sorafenib treatment could be considered as an option when patients have failed vandetanib and cabozantinib or these drugs are unavailable.


1. Castroneves LA, Negrao MV, Costa de Freitas RM, et al. Sorafenib for the treatment of progressive metastatic medullary thyroid cancer: efficacy and safety analysis [published online ahead of print December 23, 2015].Thyroid. doi:10.1089/thy.2015.0334.