Tandem high-dose chemotherapy followed by autologous transplantation in patients with locally advanced or metastatic sarcoma

Background: Patients with locally advanced or metastatic/recurrent soft tissue and Ewing's sarcoma (EWS) have few treatment options. The purpose of our phase II study was to assess the feasibility, safety and efficacy of tandem high-dose chemotherapy (HDCT) followed by autologous stem cell transplantation (ASCT) in such patients. Patients and Methods: Thirteen patients were enrolled onto this study. The first cycle of HDCT consisted of doxorubicin (150 mg/m ²) and ifosfamide (14 g/m²) mixed with mesna (14 g/m ²), while the second cycle consisted of melphalan (150 mg/m ²) and cisplatin (200 mg/m²). Results: Eleven out of 13 patients were able to complete both cycles of HDCT. No treatment-related mortality occurred and grade 3 or 4 toxicity was clinically tolerable. The 5-year progression-free survival (PFS) and overall survival (OS) for all patients was 23% (confidence interval, CI: 0-46%) and 31% (CI: 14-70%), respectively. Out of the four patients still alive, two had EWS and measurable disease at the time of ASCT and achieved a complete remission, remaining progression free 126 and 155 months after ASCT. Conclusion: Our study demonstrates the feasibility and safety of tandem HDCT in patients with high-risk or metastatic/recurrent sarcoma, with some patients achieving long-term PFS and OS.