Trofosfamide in patients with pancreatic cancer.

Background: In a previous phase II trial of ifosfamide (IFO) several patients with pancreatic cancer responded to therapy (1). Trofosfamide (TROFO) is an orally taken oxazaphosphorine prodrug. Metabolic products of TROFO include ifosfamide (IFO) and cyclophosphamide (CYCLO). In clinical pilot studies with TROFO against refractory malignancies mild toxicity was reported. Patients and methods: Since virtually all patients with advanced pancreatic cancer are symtomatic, we designed and conducted a prospective phase II trial to evaluate the activity of single agent therapy with TROFO given orally continuously (three times 50 mg daily), and to assess the toxicity, duration of remission, and overall survival of the treated patients. Results: 16 patients were enrolled onto the study. In one patient a PR has been observed; the mayor response rate was 6%. The mayor toxicity was bone marrow toxicity with granulocytopenia (WHO grade II) and anemia (WHO grade II) in virtually all patients. Conclusion: Single agent therapy with TROFO in patients with pancreatic cancer is safe and well tolerated; however, it shows low clinical activity.