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Medullary thyroid carcinoma (MTC) is a rare malignancy comprising 1-2% of all thyroid cancers in the United States. Approximately 20% of cases are familial, secondary to a germline RET mutation, while the remaining 80% are sporadic and also harbour a somatic RET mutation in more than half of all cases. Up to 15-20% of patients will present with distant metastatic disease, and retrospective series report a 10-year survival of 10-40% from time of first metastasis. Historically, systemic therapies for metastatic MTC have been limited, and cytotoxic chemotherapy has demonstrated poor objective response rates. However, in the last decade, targeted therapies, particularly multitargeted tyrosine kinase inhibitors (TKIs), have demonstrated prolonged progression-free survival in advanced and progressive MTC. Both cabozantinib and vandetanib have been approved as first-line treatment options in many countries; nevertheless, their use is limited by high toxicity rates and dose reductions are often necessary. New generation TKIs, such as selpercatinib or pralsetinib, that exhibit selective activity against RET, have recently been approved as a second-line treatment option, and they exhibit a more favourable side-effect profile. Peptide receptor radionuclide therapy or immune checkpoint inhibitors may also constitute potential therapeutic options in specific clinical settings. In this review, we aim to present all current therapeutic options available for patients with progressive MTC, as well as new or as yet experimental treatments. [ABSTRACT FROM AUTHOR]

Neuroendocrine neoplasms (NENs) are still considered to be rare neoplasms, and their epidemiology has been classically studied in large population-based cancer registries. Besides the benefits and the limitations that a cancer registry may have for all the registered cancers, the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER) Programme, has a number of drawbacks for the study of NENs. The change in management of NENs, either from diagnostic or from therapeutic points of view, the role of the Ki-67 labelling index and introduction of sensitive functional imaging, along with the misclassification of the more benign types of NENs, are the main limitations of the prognostic ability of the SEER data-base, particularly when including older data., (© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)