Abstract

BACKGROUND:

The aim of this study was to determine the response and toxicity of patients with recurrent or treatment-induced brain tumors to TMZ and oral VP-16.

METHODS:

Eleven patients with recurrent or treatment-induced malignant CNS tumors, including treatment-induced PNET (in 1 patient), brainstem glioma (in 3 patients; 1 with treatment-induced, 2 with recurrence), recurrent anaplastic astrocytoma (in 3 patients), and recurrent glioblastoma (in 4 patients) were evaluated in a pilot study of TMZ and oral VP-16 chemotherapy. All patients received TMZ at 150 mg/m2 per day on days 1 to 5 and oral VP-16 at 50 mg/m2 per day on days 1 to 12. Cycles were repeated every 28 days.

RESULTS:

None experienced major acute toxicity related to TMZ and oral VP-16 during a total of 52 treatment courses. Five (45%) of 11 patients showed a PR to treatment. Among the 11 patients enrolled, 7 patients are alive with disease at a median of 9 months from time of study entry. The 6-month PFS is 45% (95% CI, 40%-74%). The histologic subtype of the tumor, its location, and its maximum response to chemotherapy did not have an impact on the duration of disease control.

CONCLUSION:

This limited pilot study confirms the innocuousness and the activity of the combination of TMZ and oral VP-16 in recurrent malignant brain tumors. This promising activity warrants further investigation of this combination in larger phase II or III studies.