Cancer Management and Research (Aug 2019)
Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes
Abstract
Poupak Fallahi,1 Silvia Martina Ferrari,2 Giusy Elia,2 Francesca Ragusa,2 Sabrina Rosaria Paparo,2 Ilaria Ruffilli,2 Armando Patrizio,2 Gabriele Materazzi,3 Alessandro Antonelli21Department of Translational Research and of New Technologies in Medicine and Surgery, University of Pisa, Pisa, Italy; 2Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy; 3Department of Surgical, Medical, Molecular Pathology and Critical Area, University of Pisa, Pisa, ItalyCorrespondence: Alessandro AntonelliDepartment of Clinical and Experimental Medicine, School of Medicine, University of Pisa, Via Savi, 10, Pisa I-56126, ItalyTel +39 05 099 2318Fax +39 05 099 3472Email [email protected]: Medullary thyroid cancers (MTCs) are neuroendocrine tumors, which secrete calcitonin and carcinoembryonic antigen, both of which can serve as tumor markers. Extensive and accurate surgical resection is the primary treatment for MTC, whereas the use of external beam radiotherapy is limited. Moreover, since MTC is derived from thyroid parafollicular cells or C cells, it is not responsive to either radioiodine or thyroid-stimulating hormone suppression, and therefore, they cannot be considered as treatment strategies. Traditional therapies for advanced or metastatic progressive medullary thyroid cancer (pMTC) are poorly effective. Among the new approaches tested in clinical trials, targeted chemotherapies with tyrosine kinase inhibitors (TKIs) are now available and they represent effective interventions for progressive disease, with additional investigational options emerging. This paper reviews the efficacy and safety of vandetanib in patients with a pMTC, as it has been shown to improve progression-free survival (30.5 vs 19.3 months in controls). Vandetanib is approved by the FDA and EMA for symptomatic or progressive MTC in patients with unresectable locally advanced or metastatic disease in adults, adolescents, and children older than 5 years. The most common adverse events in vandetanib-treated patients are diarrhea, rash, folliculitis, nausea, QTc prolongation, hypertension, and fatigue. More data are required to deepen our knowledge on molecular biology of tumor and host defense, with the aim to achieve better prognosis and higher quality of life for affected patients.Keywords: MTC, pediatric MTC, vandetanib, RET, VEGFR, AEs
