Background: The number of cytostatic agents effective in patients with advanced soft tissue sarcoma is limited. Trofosfamide, an alkylating agent, has been shown to be effective in several solid and haematological tumors.
Patients and methods: Eighteen patients with a median age of 57 years (range, 27-78) were treated with oral trofosfamide 300 mg/d for 1 week followed by 150 mg/d given continuously to analyze the efficacy and toxicity of continuous low-dose oral trofosfamide. All had received at least one anthracycline-based chemotherapeutic regimen prior to trofosfamide.
Results: Nine patients (50%) achieved stable disease lasting for a median of 5.5+ months (range, 1-9+). The median time to progression was 10+ weeks (range, 4-37+) and the median survival 7+ months (range, 2-13+). Toxicity was mild, grade III degree toxicity was seen in 5 patients (28%): 3/2 patients with anemia/neutropenia and 2 patients with fatigue syndrome.
Conclusion: No objective remission was observed with oral low-dose trofosfamide in heavily pretreated soft tissue sarcoma patients, but almost half of the patients achieved disease stabilisation for half a year. The moderate toxicity profile observed in this study allows the consideration of trofosfamide as a reasonable palliative treatment option for patients with soft tissue sarcoma.