Cyclophosphamide for the treatment of progressive low-grade astrocytoma: A Pediatric Oncology Group Phase II study

Purpose: Results of a phase II trial of cyclophosphamide (CPM) for children with progressive low-grade astrocytoma are reported. Patients and Methods: Fifteen patients with a median age of 39 months (range, 2 to 71) were included in this study. The tumors of 11 children were located in the optic pathway, hypothalamus, or thalamus. Four courses of intravenous CPM 1.2 g/m² were administered every 3 weeks during the upfront window portion of this protocol. Subsequently, chemotherapy was to continue with CPM, vincristine, and carboplatin for 2 years. Results: By study design, the first 14 patients were centrally reviewed after completion of the initial 4 CPM courses. Toxicity was primarily hematologic. One patients had a complete response, 8 had stable disease, and 5 had progressive disease (PD). The excessive number of children with PD prompted study closure. Conclusion: CPM as used in this protocol showed insufficient activity against astrocytoma to justify further patient accrual.