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Sustained Response with Dose Reduced Selpercatinib in a Pediatric Patient with Metastatic NCOA4-RET Fusion Papillary Thyroid Carcinoma
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  • Anna C. Suydam,
  • Alicia Bach,
  • Stephanie Markovina,
  • Perry Grigsby,
  • Jennifer Sprague,
  • Amy Armstrong
Anna C. Suydam
Washington University in St Louis Department of Pediatrics
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Alicia Bach
University of Missouri School of Medicine
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Stephanie Markovina
Washington University in St Louis Washington University Physicians
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Perry Grigsby
Washington University in St Louis Washington University Physicians
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Jennifer Sprague
Washington University in St Louis Department of Pediatrics
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Amy Armstrong
Washington University in St Louis Department of Pediatrics

Corresponding Author:armstrongae@wustl.edu

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Abstract

Understanding the molecular landscape of papillary thyroid carcinoma (PTC), the most common thyroid cancer in children, creates additional therapeutic approaches. RET gene rearrangements are observed in pediatric PTC and selective inhibition of RET is now possible with specific tyrosine kinase inhibitors designed to target diverse RET-activating mutations. We present a 13-year-old female with radioactive iodine-refractory metastatic PTC, found to harbor a NCOA4-RET fusion, who responded to treatment with selpercatinib with elimination of supplemental oxygen need, marked reduction in pulmonary nodules and mediastinal lymphadenopathy, and substantial improvement in thyroglobulin levels. Response was maintained despite 2 dose reductions for possibly-related weight gain.
23 Jan 2023Submitted to Pediatric Blood & Cancer
23 Jan 2023Assigned to Editor
23 Jan 2023Submission Checks Completed
23 Jan 2023Review(s) Completed, Editorial Evaluation Pending
23 Jan 2023Reviewer(s) Assigned