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Published on: December 9, 2016
Clinical Activity of Pazopanib in Metastatic Extraosseous Ewing Sarcoma
Steven Attia1, Scott H Okuno2, Steven I Robinson2
1Mayo Clinic , Jacksonville, FL, USA.
Abstract:
We report a response to pazopanib in a 69-year-old man with heavily pre-treated metastatic extraosseous Ewing sarcoma in addition to molecular profiling of his tumor. To our knowledge, this case is the earliest to demonstrate activity of an oral multi-targeted kinase inhibitor in Ewing sarcoma. This case provides rationale for adding a Ewing sarcoma arm to SARC024, a phase II study of regorafenib, another multi-targeted kinase inhibitor, in patients with liposarcoma, osteosarcoma and Ewing and Ewing-like sarcomas (NCT02048371). This national multi-institutional study is ongoing.
Insights
A patient with metastatic extraosseous Ewing sarcoma responded to pazopanib, an oral multi-targeted kinase inhibitor. This finding supports further investigation of kinase inhibitors for Ewing sarcoma treatment.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Metastatic extraosseous Ewing sarcoma is a rare and aggressive cancer.
- Patients often receive multiple lines of treatment without durable responses.
- Novel therapeutic strategies are needed for advanced-stage disease.
Observation:
- A 69-year-old male with heavily pre-treated metastatic extraosseous Ewing sarcoma was treated with pazopanib.
- Molecular profiling of the patient's tumor was performed.
- The patient demonstrated a clinical response to pazopanib therapy.
Findings:
- This case represents the earliest documented response to an oral multi-targeted kinase inhibitor in Ewing sarcoma.
- Pazopanib exhibited anti-tumor activity in a heavily pre-treated patient.
- Tumor molecular profiling may guide targeted therapy selection.
Implications:
- This case provides a strong rationale for investigating oral multi-targeted kinase inhibitors in Ewing sarcoma.
- It supports the expansion of the SARC024 clinical trial to include a Ewing sarcoma cohort.
- Further research into pazopanib and similar agents is warranted for Ewing sarcoma treatment.

