A newborn with a large NTRK fusion positive infantile fibrosarcoma successfully treated with larotrectinib

Pediatr Blood Cancer. 2020 Sep;67(9):e28330. doi: 10.1002/pbc.28330. Epub 2020 May 26.

Abstract

Infantile fibrosarcoma (IFS) is a rare pediatric cancer that typically presents early in life. Surgical resection is commonly curative; however, resection is sometimes not possible requiring additional multimodal treatment. IFS commonly harbors a fusion in one of the neurotrophic receptor tyrosine kinase (NTRK) genes. Larotrectinib, a highly selective inhibitor of tropomyosin receptor kinase (TRK), has been shown to be well tolerated and effective in children as young as 1-month old. We report a case of IFS in a newborn treated with larotrectinib. The patient experienced a rapid clinical and radiographic response demonstrating the potential to treat newborns with larotrectinib.

Keywords: NTRK; infantile fibrosarcoma; larotrectinib; pediatric.

Publication types

  • Case Reports

MeSH terms

  • Fibrosarcoma / drug therapy*
  • Humans
  • Infant, Newborn
  • Male
  • Membrane Glycoproteins / antagonists & inhibitors
  • Membrane Glycoproteins / genetics
  • Oncogene Proteins, Fusion / genetics
  • Protein Kinase Inhibitors / therapeutic use*
  • Protein Kinases / drug effects
  • Pyrazoles / therapeutic use*
  • Pyrimidines / therapeutic use*
  • Receptor, trkA / antagonists & inhibitors
  • Receptor, trkA / genetics
  • Receptor, trkB / antagonists & inhibitors
  • Receptor, trkB / genetics
  • Receptor, trkC / antagonists & inhibitors
  • Receptor, trkC / genetics

Substances

  • Membrane Glycoproteins
  • NTRK1 protein, human
  • NTRK3 protein, human
  • Oncogene Proteins, Fusion
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • Protein Kinases
  • Receptor, trkA
  • Receptor, trkB
  • Receptor, trkC
  • tropomyosin-related kinase-B, human
  • tropomyosin kinase
  • larotrectinib