Endocrine Abstracts

Medullary thyroid carcinoma (MTC) is a rare thyroid C cell malignant tumor that accounts for 3-4% of thyroid gland neoplasias. MTC may occur sporadically or be inherited. Hereditary MTC appears as part of the MEN 2 syndrome. Germ-line mutations of the RET proto-oncogene cause hereditary cancer, whereas somatic mutations are frequently present in sporadic disease. Currently, early MTC diagnosis followed by total thyroidectomy offers the only possibility of the cure of the disease. Although the majority of MTC patients have a good prognosis, a subgroup of patients develops progressive disease and requires systemic therapy. We will focus on the current therapeutic approaches for patients with advanced disease discussing the advantages and disadvantages of molecular targeted therapies that inhibit RET and other tyrosine kinase receptors involved in tumor angiogenesis. Treatment with tyrosine kinase inhibitors (TKI) increases the progression-free survival but we still needed to answer the question of whether it impacts on overall survival. Hopefully, the cumulative knowledge about molecular profiling of MTC and the TKI-associated side effects will help in choosing the best therapeutic approach to enhance their benefits.