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Updated: May 15, 2025
Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Systemic therapies for medullary thyroid carcinoma: state of the art
Alejandro Román-González1, Ines Califano2, Marcio Concepción-Zavaleta3
1Section of Endocrinology, Department of Internal Medicine, School of Medicine, Universidad de Antioquia, (University of Antioquia), Cra. 51d No. 62-29, Medellín 050001, Colombia.
Abstract:
Medullary thyroid carcinoma (MTC) is a rare neuroendocrine tumor accounting for less than 5% of all thyroid cancers. An estimated 25% of cases are familial secondary to a germline mutation on the rearranged during transfection proto-oncogene (RET); this gene can be present as a somatic mutation in approximately 40%-60% of sporadic MTC tumors. There is an existing genotype-phenotype correlation in the clinical behavior of MTC, with the RET M918T variant associated with aggressive disease. The current systemic treatment profile for progressive metastatic MTC involves antiangiogenics multikinase inhibitors (MKI), specifically cabozantinib and vandetanib, and high-specific RET inhibitor therapy. Decisions on the timing of systemic therapy initiation in this population should involve multidisciplinary care and individualization on a case-by-case scenario; a comprehensive evaluation of performance status, tumor burden, progression rate, medical comorbidities, possible medication interactions, and goals of care must be considered in a patient-centered approach. This review summarizes the evidence on the safety, efficacy, and limitations of systemic therapies for MTC; the aim is to empower clinicians with the knowledge to optimally manage patients with advanced, progressive, or metastatic MTC.
Insights
Medullary thyroid carcinoma (MTC) is a rare cancer. This review details systemic therapies, including multikinase inhibitors and RET inhibitors, for advanced MTC, emphasizing personalized patient care.
Area of Science:
- Oncology
- Genetics
- Endocrinology
Background:
- Medullary thyroid carcinoma (MTC) is a rare neuroendocrine tumor, comprising <5% of thyroid cancers.
- Germline or somatic mutations in the rearranged during transfection (RET) proto-oncogene are implicated in MTC development.
- The RET M918T variant is linked to aggressive MTC disease, highlighting genotype-phenotype correlations.
Purpose of the Study:
- To review the safety, efficacy, and limitations of current systemic therapies for MTC.
- To provide clinicians with knowledge for optimal management of advanced MTC.
- To emphasize a patient-centered approach in treatment decisions.
Main Methods:
- Literature review of systemic therapies for MTC.
- Analysis of current treatment guidelines and clinical trial data.
- Synthesis of evidence on multikinase inhibitors (MKIs) and RET inhibitors.
Main Results:
- Current systemic treatments for progressive metastatic MTC include MKIs (cabozantinib, vandetanib) and targeted RET inhibitors.
- Treatment decisions require individualized, multidisciplinary evaluation considering patient status and disease characteristics.
- Evidence on safety, efficacy, and limitations of these therapies is summarized.
Conclusions:
- Optimal management of advanced MTC necessitates a comprehensive, patient-centered approach.
- Understanding treatment options and limitations empowers clinicians in managing MTC.
- Personalized therapy selection is crucial for patients with advanced, progressive, or metastatic MTC.
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