• Joseph Chao, G Thomas Budd, Peiguo Chu, Paul Frankel, Dolores Garcia, Maribel Junqueira, Sofia Loera, George Somlo, Judith Sato, and Warren A Chow.
• Department of Medical Oncology, City of Hope Comprehensive Cancer Center, 1500 E. Duarte Road, Duarte, CA 91010, USA. jchao@coh.org
• Anticancer Res. 2010 Feb 1; 30 (2): 547-52.

BackgroundWe have previously shown that the receptor tyrosine kinases, KIT and PDGFRalpha, are expressed on ESFT cell lines, and that imatinib induces dose-dependent apoptosis (1). We conducted a Phase II trial to evaluate the effectiveness of imatinib for patients with recurrent ESFT or DSRCT expressing KIT and/or PDGFRalpha.Patients And MethodsPatients were selected for tumor immunohisto-chemical expression > or =2+/4+ for KIT or PDGFRalpha. Imatinib was administered orally 400 mg twice/day for 28 days/course. Primary endpoint was response.ResultsSeven patients were enrolled and evaluated. One patient with 3+/4+ PDGFRalpha and 3+/4+ KIT expression had a partial response through 8 courses. 4 patients had progression after 1 cycle. Two patients were not evaluable due to one early death and one refusing treatment.ConclusionThis study intended to enrich for molecular factors that potentially predict response. Given the poor prognosis with recurrent ESFT, further studies with other novel KIT and PDGFRalpha inhibitors are needed.

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