High-dose cyclophosphamide chemotherapy for recurrent CNS tumors in children

Abstract

A phase II chemotherapy trial was conducted in 18 children with recurrent brain tumors, using high doses (80 mg/kg or greater) of i.v. cyclophosphamide. All 8 patients with medulloblastomas responded; 2 patients with systemic metastases had complete responses and 6 others had partial responses. In 7 patients with gliomas, there were 1 complete and 4 partial responses. In a 3rd group, all 3 patients with intracranial germ-cell tumors had partial responses. The overall response rate was, therefore, 89% (16 of 18 patients) and the mean duration of response was 7 mo. (range 2-24 or more mo.). The hematological toxicity was considerable, with 2 deaths possibly related to chemotherapy: 1 patient, a recipient of unirradiated packed cells, died from a graft vs. host reaction and the other died from an intracranial hemorrhage during a thrombocytopenic episode. Four patients had prior chemotherapy; and 10 patients had prior neuraxis radiation therapy. These patients tolerated aggressive chemotherapy reasonably well. The results are sufficiently encouraging to justify a phase III trial in patients with newly diagnosed disease.