Tamoxifen and Carboplatin for Children With Low-Grade Gliomas: A Pilot Study at St. Jude Children's Research Hospital

J Pediatr Hematol Oncol. May-Jun 2000;22(3):247-51. doi: 10.1097/00043426-200005000-00010.


Purpose: The authors conducted a single-arm, prospective study using tamoxifen and carboplatin for the treatment of children with progressive or symptomatic 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 because of the diagnosis of a nonmalignant condition. Patients were treated with daily tamoxifen (20 mg/m2 administered twice per day) in addition to targeted, monthly intravenous carboplatin at an area under the curve (AUC) exposure of 6.5 mg/mL x minute for 1 year or until they had clinical or radiologic evidence of disease progression.

Results: The median age at diagnosis was 5.3 years, the median age at initiation of chemotherapy was 8.3 years. Eight patients had tumors of the hypothalamus/optic pathway, two patients had thalamic tumors, and one patient each had tumors in the temporal lobe, tectum, and brain stem. Tumor histologic findings included fibrillary astrocytoma (n = 2), juvenile pilocytic astrocytoma (n = 6), and oligodendroglioma (n = 1). The best response to therapy 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 carboplatin chemotherapy did not result in a significant number of objective responses in children with low-grade gliomas. The progression-free survival is similar to that of other published series. Nonmyelosuppressive agents such as tamoxifen deserve additional evaluation in the treatment of children with low-grade gliomas.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Astrocytoma / drug therapy*
  • Astrocytoma / mortality
  • Brain Neoplasms / drug therapy*
  • Brain Neoplasms / mortality
  • Carboplatin / administration & dosage
  • Child
  • Child, Preschool
  • Disease Progression
  • Disease-Free Survival
  • Enzyme Inhibitors / administration & dosage
  • Female
  • Humans
  • Life Tables
  • Male
  • Oligodendroglioma / drug therapy*
  • Oligodendroglioma / mortality
  • Prospective Studies
  • Protein Kinase C / antagonists & inhibitors
  • Survival Analysis
  • Survival Rate
  • Tamoxifen / administration & dosage
  • Treatment Outcome


  • Enzyme Inhibitors
  • Tamoxifen
  • Carboplatin
  • Protein Kinase C