Background: Chiasmatic-hypothalamic gliomas are not amenable to surgical resection and therefore are treated with either radiotherapy or chemotherapy. Etoposide (VP-16), administered on a long-term oral schedule, represents a novel chemotherapeutic approach.
Patients and methods: Fourteen patients (age range, 2 to 15 years) were treated with etoposide following tumor progression as determined by clinical and neuroradiographic examinations. Thirteen patients had received prior radiotherapy, and 12 received prior nitrosourea-based chemotherapy. Etoposide was administered orally; each cycle consisted of 50 mg/m2 per day on days 1 to 21 and days 36 to 57. Clinical and neuroradiographic examinations were performed from days 58 to 72 prior to the start of each cycle of therapy. Complete blood cell counts were performed weekly.
Results: Treatment-related complications included partial alopecia (n = 7), diarrhea (n = 6), weight loss (n = 5), neutropenia (n = 4), and thrombocytopenia (n = 4). Three patients required a transfusion (ie, red blood cell [n = 3] and platelet [n = 2] transfusions), and one patient required antibiotic treatment of neutropenic fever. There were no treatment-related deaths. Fourteen patients were evaluable; in eight of these 14 patients, a response was demonstrated radiographically (complete response [n = 1], partial response [n = 4], and stable disease [n = 3]), with a median duration of response of 8 months.
Conclusions: Long-term treatment with oral etoposide was well tolerated by the patients in this study, and etoposide was a relatively nontoxic chemotherapeutic agent with apparent activity in this small cohort of patients who had recurrent chiasmatic-hypothalamic gliomas.