Infantile Fibrosarcoma With NTRK3–ETV6 Fusion Successfully Treated With the Tropomyosin-Related Kinase Inhibitor LOXO-101

Ramamoorthy Nagasubramanian*, Julie Wei, Paul Gordon, Jeff C. Rastatter, Michael C. Cox, Alberto Pappo

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

73 Scopus citations

Abstract

Infantile fibrosarcoma (IFS) is a rare pediatric cancer typically presenting in the first 2 years of life. Surgical resection is usually curative and chemotherapy is active against gross residual disease. However, when recurrences occur, therapeutic options are limited. We report a case of refractory IFS with constitutive activation of the tropomyosin-related kinase (TRK) signaling pathway from an ETS variant gene 6–neurotrophin 3 receptor gene (ETV6–NTRK3) gene fusion. The patient enrolled in a pediatric Phase 1 trial of LOXO-101, an experimental, highly selective inhibitor of TRK. The patient experienced a rapid, radiographic response, demonstrating the potential for LOXO-101 to provide benefit for IFS harboring NTRK gene fusions.

Original languageEnglish (US)
Pages (from-to)1468-1470
Number of pages3
JournalPediatric Blood and Cancer
Volume63
Issue number8
DOIs
StatePublished - Aug 1 2016

Keywords

  • ETV6–NTRK3
  • LOXO-101
  • gene fusion
  • infantile fibrosarcoma
  • tropomyosin-related kinase (TRK)

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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