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OER-073: A multicenter phase 2 study evaluating the role of pazopanib in angiosarcoma
Julio Alvarenga Thiebaud1, Vinod Ravi2, Samuel Litwin3
1Department of Hematology Oncology, Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA.
Background:
Angiosarcomas are rare mesenchymal sarcomas that can present as primary cutaneous or noncutaneous disease. They express a variety of vascular endothelial growth factor receptors. The authors hypothesized that the treatment of angiosarcoma with pazopanib, a multikinase inhibitor with activity against vascular endothelial growth factor receptors, would result in disease response and prolonged disease stabilization.
Methods:
This was an open-label, phase 2 trial of pazopanib in patients who had incurable angiosarcoma. The co-primary end points were response according to the Response Evaluation Criteria in Solid Tumors and progression-free survival (PFS) at 3 months. The starting dose of pazopanib was 800 mg daily.
Results:
Twenty-nine patients were accrued between 2011 and 2018, and 22 patients were evaluable for response. Toxicities were similar to those identified in prior reports. There was one partial response (3%), and the clinical benefit rate (including complete responses, partial responses, and stable disease) was 48%, which was observed more frequently in patients who had cutaneous disease. The median PFS was 14.4 weeks, and the 3-month PFS rate determined by Kaplan-Meier estimate was 54.6% (95% CI, 36.0%-82.9%), meeting the primary study objective. The Kaplan-Meier overall survival estimate was 16.1 months.
Conclusions:
Pazopanib therapy in patients who had incurable angiosarcoma was associated with meaningful disease control, especially in those who had cutaneous disease with limited objective responses.
Lay Summary:
Angiosarcoma is a rare cancer that can be found on the skin or in internal organs. This study tested pazopanib, an oral targeted medication, to determine its benefit in patients with angiosarcoma who could not undergo the removal of their tumors by surgery. Pazopanib treatment was safe, and no new side effects were reported. The study showed that pazopanib controlled tumor growth in one half of patients at 3 months and was more common in angiosarcomas of the skin; it led to tumor shrinkage in a minority of patients (1 of 29).
Insights
Pazopanib demonstrated disease control in patients with incurable angiosarcoma, particularly those with cutaneous disease. The treatment showed a 48% clinical benefit rate, with limited objective responses observed.
Area of Science:
- Oncology
- Medical Research
- Pharmacology
Background:
- Angiosarcomas are rare mesenchymal tumors with vascular endothelial growth factor receptor expression.
- Pazopanib, a multikinase inhibitor, targets vascular endothelial growth factor receptors.
Purpose of the Study:
- To evaluate the efficacy of pazopanib in patients with incurable angiosarcoma.
- To assess disease response and progression-free survival (PFS) at 3 months.
Main Methods:
- An open-label, phase 2 clinical trial was conducted.
- Patients received pazopanib at a starting dose of 800 mg daily.
- Co-primary endpoints included Response Evaluation Criteria in Solid Tumors response and 3-month PFS.
Main Results:
- A clinical benefit rate of 48% was observed in 22 evaluable patients.
- A partial response was seen in 3% of patients; higher benefit in cutaneous disease.
- Median PFS was 14.4 weeks, with a 3-month PFS rate of 54.6%.
Conclusions:
- Pazopanib therapy provides meaningful disease control in incurable angiosarcoma.
- Efficacy, particularly disease stabilization, was more pronounced in patients with cutaneous angiosarcoma.
