Clinical and radiographic response in three children with recurrent malignant cerebral tumors with high-dose tamoxifen

The purpose of this study was to deliver tamoxifen as a antiangiogenic ther apy to children with recurrent progressive malignant brain tumors. Tamoxife n was administered orally in very high dosage to one child as monotherapy a nd to two children in combination with oral etoposide and dexamethasone. On e boy was diagnosed with high-grade astrocytoma in the brain stem, one girl with anaplastic ependymoma of the fourth ventricule, and one girl with hig h-grade astrocytoma in the midbrain. Conventional treatment with multiple s urgeries, first- and second-line chemotherapy, and external beam therapy ha d failed. Tumor reduction was seen I radiographic images together with clin ical Improvement in 2 children, and clinical and radiographic halting of tu mor progression tumor progression was demonstrated in the patient with anap lastic ependymoma. None of the patients developed complications from the tr eatment. Follow up of the patients ranged from 15 to 30 months with a mean of 17 months. These encouraging preliminary results suggest a potential for this type of therapy. More studies are needed to start clinical trials and prove that angiostatic activity may contribute to the therapeutic effect o f antiestrogens in estrogen receptor-negative tumors.