Chronic oral VP-16 for recurrent medulloblastoma

Chronic oral VP-16 (Etoposide) is a chemotherapy regimen with wide application in oncology and documented efficacy against germ cell tumors, lymphomas, Kaposi sarcoma, and glial brain tumors. Eight patients ranging in age from 4 to 36 years (median 7.5 years) with locally recurrent medulloblastoma were treated with VP-16. No patient displayed evidence of cerebrospinal fluid dissemination, distant brain or spine parenchymal metastases, or extraneural metastatic disease. All patients had previously been treated with surgery (gross total resection, 5; subtotal resection, 3), craniospinal radiotherapy, and platinum-based chemotherapy (adjuvant, 3; salvage, 8). Each cycle of therapy consisted of 21 days of VP-16 (50 mg/m2/day) followed by a 7 to 14 day rest followed by an additional 21 days of VP-16 (50 mg/m2/day). Complete blood counts were obtained weekly. Neurologic examination and brain magnetic resonance imaging scan with contrast were performed prior to each cycle of therapy. Treatment-related complications included: partial alopecia (5 patients); diarrhea (4); weight loss (3); anemia (2); neutropenia (4); and thrombocytopenia (4). Two patients required transfusion and 1 patient received antibiotics for neutropenic fever. All patients were evaluable for response: 3 demonstrated progressive disease after the first cycle of VP-16, 3 had stable disease (range 4 to 6 months) and 2 had partial neuroradiographic responses (8 and 10 months). Median duration of response and stable disease was 6 months (range: 4 to 10 months) in 5 of 8 (62.5%) patients. Chronic oral VP-16 is a well-tolerated and relatively non-toxic chemotherapeutic agent with demonstrated activity in locally recurrent medulloblastoma.