• Yelda Jozaghi, Mark Zafereo, Michelle D Williams, Maria K Gule-Monroe, Jennifer Wang, Elizabeth G Grubbs, Ara Vaporciyan, Mimi I Hu, Naifa Busaidy, Ramona Dadu, Steven G Waguespack, Vivek Subbiah, and Maria Cabanillas.
• Department of Head and Neck Surgery, The University of Texas M. D. Anderson Cancer Center, Houston, Texas, USA.
• Head Neck. 2021 Jan 1; 43 (1): E7-E12.

BackgroundTargeted kinase inhibitors have been increasingly utilized in the treatment of advanced medullary thyroid cancer (MTC) over the last decade. Recently, highly potent next generation selective RET inhibitors have been clinically validated, and selpercatinib was recently Food and Drug Administration (FDA)-approved for advanced MTC. The advent of highly selective, potent RET inhibitors is broadening the treatment options for patients with RET-mutated cancers.MethodsWe report the first published case of neoadjuvant selpercatinib followed by surgery for a patient with initially unresectable, widely metastatic, RET-mutated MTC who was treated on a single patient protocol.ResultsAfter greater than 50% RECIST response, the patient underwent complete surgical resection followed by selpercatinib resumption. He remains locoregionally disease-free 21 months after starting therapy with stable metastatic disease (after initial partial response); and calcitonin/CEA continue to decline.ConclusionThis novel treatment strategy for locoregionally advanced RET-mutated MTC warrants further study in clinical trials.© 2020 The Authors. Head & Neck published by Wiley Periodicals LLC.

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