Clinical Activity of Pazopanib in Metastatic Extraosseous Ewing Sarcoma.
Bottom Line: 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 national multi-institutional study is ongoing.
Affiliation: Mayo Clinic , Jacksonville, FL, USA.
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.
No MeSH data available.
Related in: MedlinePlus
Mentions: In early 2011, several months after finishing radiation, new, innumerable bilateral pulmonary nodules were noted on CT. Transbronchial biopsy was histologically consistent with metastasis from Ewing sarcoma (Figure 1D,E). FISH indicated rearrangement involving the EWSR1 gene region. He received cyclophosphamide and topotecan with disease stability noted after two cycles. After six cycles, enlarging lung, new liver and new diffuse sclerotic bony lesions were noted. He then received irinotecan with temozolomide. After two cycles, a dramatic response at all disease sites was noted. Over 10 four-week cycles, spanning a year with treatment breaks, he developed a near complete response with only several subcentimeter bilateral lung nodules remaining. Platelet counts on temozolomide ranged between 50,000 and 75,000. After 10 cycles, the lung nodules began to enlarge minimally (Figure 2). He received another three cycles during which time the lung nodules progressed. This regimen was stopped. He had exhausted all standard treatment lines for Ewing sarcoma, and was not eligible for a clinical study due to thrombocytopenia. Molecular testing on tissue retrieved prior to treatment with temozolomide and irinotecan (Caris, Table 1) was ordered at the patient’s request. The test suggested that only temozolomide and dacarbazine had possible clinical benefit by virtue of loss of O-6-methylguanine DNA methyltransferase expression (Table 1).
No MeSH data available.