Medullary Thyroid Cancer: Updates and Challenges

作者：Matti L. Gild, Roderick John Clifton-Bligh, Lori J. Wirth, Bruce G. Robinson · 发表于：Endocrine Reviews · 年份：2023 · DOI：10.1210/endrev/bnad013 · 被引用次数：107 · 研究领域：Thyroid Cancer Diagnosis and Treatment、Cancer-related Molecular Pathways、BRCA gene mutations in cancer

A personalized approach to the management of medullary thyroid cancer (MTC) presents several challenges; however, in the past decade significant progress has been made in both diagnostic and treatment modalities. Germline rearranged in transfection (RET) testing in multiple endocrine neoplasia 2 and 3, and somatic RET testing in sporadic MTC have revolutionized the treatment options available to patients. Positron emission tomography imaging with novel radioligands has improved characterization of disease and a new international grading system can predict prognosis. Systemic therapy for persistent and metastatic disease has evolved significantly with targeted kinase therapy especially for those harboring germline or somatic RET variants. Selpercatinib and pralsetinib are highly selective RET kinase inhibitors that have shown improved progression-free survival with better tolerability than outcomes seen in earlier multikinase inhibitor studies. Here we discuss changes in paradigms for MTC patients: from determining RET alteration status upfront to novel techniques for the evaluation of this heterogenous disease. Successes and challenges with kinase inhibitor use will illustrate how managing this rare malignancy continues to evolve.