Prolonged response to sorafenib in a patient with refractory metastatic osteosarcoma and a somatic PDGFRA D846V mutation

Amy E. Armstrong*, David O. Walterhouse, Patrick J. Leavey, Jennifer Reichek, Amy L. Walz

*Corresponding author for this work

Research output: Contribution to journalArticle

1 Scopus citations


Outcome for patients with metastatic or recurrent/refractory osteosarcoma remains poor. Responses to sorafenib, a multikinase inhibitor, have been seen in recurrent/refractory osteosarcoma, although specific biomarkers of response have not been described. We report a partial response in a 7-year-old with refractory osteosarcoma treated with sorafenib 200 mg twice daily. Toxicities included Common Terminology Criteria for Adverse Events Grade 2 skin toxicities and growth suppression. After 51 months of therapy, he suffered a recurrence. Tumor sequencing later revealed a PDGFRA D846V mutation that was not identified in the relapse specimen. This case demonstrates prolonged partial response to sorafenib and provides a potential biomarker for response.

Original languageEnglish (US)
Article numbere27493
JournalPediatric Blood and Cancer
Issue number1
StateAccepted/In press - Jan 1 2018



  • osteosarcoma
  • platelet-derived growth factor receptor (PDGFR)
  • sorafenib

ASJC Scopus subject areas

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

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