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Refractory infantile high-grade glioma containing TRK-fusion responds to larotrectinib
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  • Torin Waters,
  • Steven Moore,
  • Yutaka Sato,
  • Brian Dlouhy,
  • Mariko Sato
Torin Waters
University of Iowa
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Steven Moore
University of Iowa
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Yutaka Sato
University of Iowa
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Brian Dlouhy
University of Iowa
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Mariko Sato
University of Iowa Children's Hospital
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Abstract

High-grade gliomas (HGGs) are a challenging group of brain tumors. Identification of targetable genetic alterations such as TRK fusions are providing new treatment options. We discuss an infant who presented with hydrocephalus and a cystic mass at birth, while a solid tumor component of the mass was only appreciated later. Tumor analysis was consistent with HGG containing a TRK fusion. Despite treatment with conventional chemotherapy, his tumor progressed in size. He was started on larotrectinib and experienced a significant decrease in tumor burden. This case demonstrates that targeted therapy may be effective for a subset of TRK fusion-positive CNS tumors.