Recurrent central nervous system medulloepithelioma: Response and outcome following marrow-ablative chemotherapy with stem cell rescue

Medulloepithelioma is a rare primitive neuroectodermal tumor of the central nervous system usually developing in childhood, displaying highly malignant behavior, with early progression or recurrence. Once a recurrence develops, death reportedly follows with invariable rapidity. The purpose of this study was to evaluate the efficacy of high-dose, marrow-ablative chemotherapy with autologous hemopoietic stem cell rescue in the treatment of recurrent central nervous system medulloepithelioma. Three young children with recurrent central nervous system medulloepithelioma received high-dose marrow-ablative chemotherapy with thiotepa and etoposide either alone (one patient) or with the addition of carboplatin (two patients). This was followed by irradiation only in one patient. One child with residual radiographic tumor at the time of treatment could be evaluated for response and showed complete resolution of leptomeningeal disease after receiving marrow-ablative chemotherapy. Two children developed tumor recurrence at 2.0 and 5.5 months after receiving marrow-ablative chemotherapy. The third child continues free of tumor beyond 12 years from treatment. The authors' experience with marrow-ablative chemotherapy and autologous hemopoietic stem cell rescue suggests that this treatment strategy might be beneficially incorporated into the initial treatment approach for young children with medulloepithelioma.