Prolonged response to sorafenib in a patient with refractory metastatic osteosarcoma and a somatic PDGFRA D846V mutation.
Pediatr Blood Cancer
; 66(1): e27493, 2019 01.
Article
em En
| MEDLINE
| ID: mdl-30318721
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.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Neoplasias Ósseas
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Biomarcadores Tumorais
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Osteossarcoma
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Resistencia a Medicamentos Antineoplásicos
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Receptor alfa de Fator de Crescimento Derivado de Plaquetas
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Sorafenibe
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Mutação
Tipo de estudo:
Prognostic_studies
Limite:
Child
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Humans
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Male
Idioma:
En
Ano de publicação:
2019
Tipo de documento:
Article