Efficacy of temozolomide in a central nervous system relapse of neuroblastoma with O 6 -methylguanine methyltransferase (MGMT) promoter methylation.

We describe a case of a 5-year-old girl with central nervous system relapse of neuroblastoma after high-dose chemotherapy and autologous stem cell transplantation. Although the brain metastasis was surgically removed, she had a second relapse in the same region with leptomeningeal dissemination despite receiving irinotecan. Administration of temozolomide in addition to irinotecan led to her third complete response and the patient has been in complete response for >24 months. The tumor had no expression of the O -methylguanine methyltransferase (MGMT) gene due to promoter methylation. Temozolomide is an attractive candidate treatment in neuroblastoma with methylated MGMT, especially in central nervous system relapsed cases.