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Medullary thyroid cancer: Chemotherapy and immunotherapy

Steven I Sherman, MD
Section Editor
Douglas S Ross, MD
Deputy Editor
Jean E Mulder, MD


Medullary thyroid cancers (MTCs) are neuroendocrine tumors of thyroid parafollicular cells that do not concentrate iodine. They occur both as sporadic tumors and as components of multiple endocrine neoplasia (MEN) type 2. They secrete calcitonin and carcinoembryonic antigen (CEA), both of which can serve as tumor markers. (See "Medullary thyroid cancer: Clinical manifestations, diagnosis, and staging".)

The primary treatment for MTC is extensive and meticulous surgical resection. There is a limited role for external beam radiotherapy. Because the neuroendocrine-derived MTC is not responsive to either radioiodine or thyroid-stimulating hormone (TSH) suppression, these options are not available for treatment of progressive metastatic MTC. (See "Medullary thyroid cancer: Treatment and prognosis".)

Patients with progressive or symptomatic metastatic disease who cannot be treated by surgery or radiotherapy should be considered candidates for systemic therapy. New approaches based upon application of targeted chemotherapies are now available as effective interventions for progressive disease, with additional investigational options emerging. Alternatively, treatment with either cytotoxic chemotherapy or biologic response modifiers may provide some benefit for occasional patients who fail or are ineligible for targeted therapies.

Current and experimental chemotherapies for advanced medullary thyroid carcinomas will be reviewed here. Chemotherapies for differentiated and anaplastic thyroid carcinomas are discussed separately. (See "Differentiated thyroid cancer refractory to standard treatment: Chemotherapy" and "Anaplastic thyroid cancer".)


As in other tumors, tyrosine kinases function in MTC to stimulate tumor proliferation, angiogenesis, invasion, and metastasis. Small molecule inhibitors of select tyrosine kinases have been of interest for the treatment of advanced MTC, given the oncogenic role of inherited and somatic mutations in the tyrosine kinase RET, as well as the contributory roles of tyrosine kinases in growth factor receptors such as the vascular endothelial growth factor receptor (VEGFR) [1,2]. These drugs partially inhibit multiple kinases at nanomolar concentrations and often affect multiple signaling pathways. (See "Classification and genetics of multiple endocrine neoplasia type 2" and "Overview of angiogenesis inhibitors", section on 'Small molecule tyrosine kinase inhibitors'.)

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Literature review current through: Nov 2017. | This topic last updated: Sep 11, 2017.
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