Publications

Synopsis: This multidisciplinary expert panel convened under the American Head and Neck Society Endocrine Surgery Section together with ITOG to establish consensus definitions and best practices for mutational testing in advanced thyroid cancer. Through a structured Delphi process and extensive literature review, the group generated 20 evidence-based statements outlining the molecular characteristics that define advanced disease, when and how somatic genetic testing should be applied, and how molecular profiles can guide targeted therapy decision-making in patients with advanced thyroid cancer. This consensus reflects evolving systemic treatment options and aims to standardize how advanced disease is characterized and managed using molecular diagnostics.

Synopsis: This international, phase II study evaluated lenvatinib, a multi-kinase inhibitor, in patients with anaplastic thyroid cancer (ATC), an aggressive and highly lethal form of thyroid carcinoma with few effective systemic options. Patients received daily oral lenvatinib and were assessed for tumor response, safety, and survival outcomes. The trial sought to determine whether lenvatinib could induce objective responses and acceptable safety in a disease setting with limited treatment efficacy. While the study faced challenges inherent to rare aggressive cancers, it provides important insights on the role and tolerability of lenvatinib in ATC and underscores the continued need for effective therapies in this population.

Synopsis: In this landmark phase 1/2 trial, selpercatinib, a selective RET inhibitor, was tested in patients with RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers. The study demonstrated high and durable tumor response rates across cohorts, including patients previously treated with other kinase inhibitors. Tumor regression and durable disease control were seen in the majority of RET-altered cases, and the drug was generally well-tolerated, with mostly low-grade side effects. These results helped support regulatory approvals for selpercatinib in RET-driven thyroid cancers and highlighted the importance of molecular profiling to identify patients who may benefit from targeted therapy.

Synopsis: This multicenter, phase II ITOG trial assessed cabozantinib, a multi-targeted tyrosine kinase inhibitor, as salvage therapy in patients with radioiodine-refractory differentiated thyroid cancer (DTC) that had progressed despite prior VEGFR-targeted therapy. In this hard-to-treat population, cabozantinib produced meaningful objective tumor responses and disease control, with a median progression-free survival suggesting clinical benefit. The study contributed evidence supporting the effectiveness of cabozantinib in overcoming resistance to earlier kinase inhibitors and helped inform clinical decision-making in refractory DTC.

Synopsis: This position statement from ITOG articulated the group’s perspective on the role of correlative science embedded within clinical trials. It emphasizes the importance of integrating biomarker analysis, genomic profiling, and translational endpoints into prospective studies to better understand mechanisms of response and resistance, optimize trial design, and accelerate discovery. By advocating standardized approaches to biospecimen collection and molecular interrogation across trials, the statement supports stronger scientific integration between clinical outcomes and biological insights.