Tamoxifen and carboplatin for children with low-grade gliomas: A pilot study at St. Jude Children's Research Hospital

Purpose: The authors conducted a single-arm, prospective study using tamoxi fen and carboplatin for the treatment of children with progressive or sympt omatic low-grade gliomas. Patients and Methods: Fourteen children with consecutively diagnosed cases of low-grade glioma were enrolled in this study; all patients were younger than 14 years. One patient was excluded after induction chemotherapy becaus e of the diagnosis of a nonmalignant condition. Patients were treated with daily tamoxifen (20 mg/m(2) administered twice per day) in addition to targ eted, monthly intravenous carboplatin at an area under the curve (AUC) expo sure of 6.5 mg/mL x minute for 1 year or until they had clinical or radiolo gic evidence of disease progression. Results: The median age at diagnosis was 5.3 years, the median age at initi ation of chemotherapy was 8.3 years. Eight patients had tumors of the hypot halamus/optic pathway, two patients had thalamic tumors, and one patient ea ch had tumors in the temporal lobe, tectum, and brain stem. Tumor histologi c findings included fibrillary astrocytoma (n = 2), juvenile pilocyric astr ocytoma (n = 6), and oligodendroglioma (n = 1). The best response to therap y was a partial response in two patients, stable disease in nine patients, and progressive disease in two patients. The overall survival at 3 years is 69%. The 3-year progression-free survival is 47%. Tamoxifen and carboplati n chemotherapy did not result in a significant number of objective response s in children with low-grade gliomas. The progression-free survival is simi lar to that of other published series. Nonmyelosuppressive agents such as t amoxifen deserve additional evaluation in the treatment of children with lo w-grade gliomas.