The safety and efficacy of vandetanib in the treatment of progressive medullary thyroid cancer.

INTRODUCTION: Traditional therapies for advanced or metastatic progressive medullary thyroid cancer (pMTC) are poor effective. Several TKIs have been tested in clinical trials in pMTC patients. Areas covered: This paper reviews efficacy and safety of vandetanib in the treatment of pMTC. Expert commentary: Vandetanib (trade name CAPRELSA® [Vandetanib]) has been shown to improve progression-free survival (30.5 vs 19.3 months in controls) in pMTC patients. Vandetanib is approved by FDA and EMA for metastatic MTC in adults; in adolescents and children with metastatic or locally advanced MTC, vandetanib seems to be effective. The most common adverse events in vandetanib-treated patients are: diarrhea, rash, folliculitis, nausea, QTc prolongation, hypertension and fatigue. In patients with aggressive differentiated thyroid cancer, vandetanib has shown promising results. Further research is needed to determine the ideal targeted therapy, based on tumor molecular characterization and host factors, to obtain the best response in terms of survival and quality of life.