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Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes

Authors Fallahi P, Ferrari SM, Elia G, Ragusa F, Paparo SR, Ruffilli I, Patrizio A, Materazzi G, Antonelli A

Received 30 March 2019

Accepted for publication 23 July 2019

Published 21 August 2019 Volume 2019:11 Pages 7893—7907

DOI https://doi.org/10.2147/CMAR.S127848

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Amy Norman

Peer reviewer comments 2

Editor who approved publication: Dr Ahmet Emre Eskazan


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 Antonelli2

1Department 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, Italy

Correspondence: Alessandro Antonelli
Department of Clinical and Experimental Medicine, School of Medicine, University of Pisa, Via Savi, 10, Pisa I-56126, Italy
Tel +39 05 099 2318
Fax +39 05 099 3472
Email alessandro.antonelli@med.unipi.it

Abstract: 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

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