Abstract Anaplastic thyroid cancer (ATC) is a rare and aggressive tumor with a historical median survival of less than six months. Most patients have advanced disease at the time of diagnosis. Traditional cytotoxic chemotherapy has not shown survival benefits. Molecular testing and next-generation sequencing are used to identify specific targets for patients with ATC. If positive for BRAF V600E mutation, patients can be treated with BRAF/MEK inhibitors. Other therapies include tyrosine kinase inhibitors (e.g., lenvatinib) and recently immune checkpoint inhibitors which have shown remarkable results in a subset of patients, including when used along with targeted therapy. An illustrative case of a patient with advanced BRAF -mutant ATC (respiratory failure on a ventilator) who recovered with a combination of the anti-PD1 nivolumab and the BRAF V600E targeted drug vemurafenib is shown. Further studies using biomarker-based gene- and immune-targeted agents, including in combination, are needed in ATC.
Khalid et al. (Wed,) studied this question.
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