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

Research output: Contribution to journalArticle

Abstract

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
DOIs
StateAccepted/In press - Jan 1 2018

Fingerprint

Osteosarcoma
Mutation
Biomarkers
Recurrence
Terminology
Skin
Growth
sorafenib
Neoplasms
Therapeutics

Keywords

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

ASJC Scopus subject areas

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

Cite this

Prolonged response to sorafenib in a patient with refractory metastatic osteosarcoma and a somatic PDGFRA D846V mutation. / Armstrong, Amy E.; Walterhouse, David O.; Leavey, Patrick J.; Reichek, Jennifer; Walz, Amy L.

In: Pediatric Blood and Cancer, 01.01.2018.

Research output: Contribution to journalArticle

@article{b4d1612d0a144a538c0c7e7e34825f58,
title = "Prolonged response to sorafenib in a patient with refractory metastatic osteosarcoma and a somatic PDGFRA D846V mutation",
abstract = "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.",
keywords = "osteosarcoma, platelet-derived growth factor receptor (PDGFR), sorafenib",
author = "Armstrong, {Amy E.} and Walterhouse, {David O.} and Leavey, {Patrick J.} and Jennifer Reichek and Walz, {Amy L.}",
year = "2018",
month = "1",
day = "1",
doi = "10.1002/pbc.27493",
language = "English (US)",
journal = "Pediatric Blood and Cancer",
issn = "1545-5009",
publisher = "Wiley-Liss Inc.",

}

TY - JOUR

T1 - Prolonged response to sorafenib in a patient with refractory metastatic osteosarcoma and a somatic PDGFRA D846V mutation

AU - Armstrong, Amy E.

AU - Walterhouse, David O.

AU - Leavey, Patrick J.

AU - Reichek, Jennifer

AU - Walz, Amy L.

PY - 2018/1/1

Y1 - 2018/1/1

N2 - 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.

AB - 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.

KW - osteosarcoma

KW - platelet-derived growth factor receptor (PDGFR)

KW - sorafenib

UR - http://www.scopus.com/inward/record.url?scp=85054905863&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85054905863&partnerID=8YFLogxK

U2 - 10.1002/pbc.27493

DO - 10.1002/pbc.27493

M3 - Article

C2 - 30318721

AN - SCOPUS:85054905863

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

SN - 1545-5009

M1 - e27493

ER -