• Halldora K Thorarinsdottir, Brian Rood, Naynesh Kamani, Debbie Lafond, Evelio Perez-Albuerne, Brett Loechelt, Roger J Packer, and Tobey J MacDonald.
• Division of Hematology and Oncology, Children's National Medical Center, Washington, District of Columbia, USA.
• Pediatr Blood Cancer. 2007 Mar 1; 48 (3): 278-84.

BackgroundChildren <4 years of age (yo) with malignant central nervous system (CNS) tumors have a dismal prognosis. In an attempt to delay or obviate radiation therapy (XRT) and improve outcome, our institution has treated children <4 yo with newly diagnosed malignant CNS tumors with high-dose chemotherapy (HDC) and autologous stem cell rescue (ASCR) followed by selective XRT.ProcedureFifteen children (age 4-38 months) with malignant CNS tumors have completed treatment with HDC/ASCR. All patients received three cycles of induction chemotherapy (cisplatin 3.5 mg/kg- day 0, cyclophosphamide 60 mg/kg- day 1 and 2, etoposide 2.5 mg/kg- day 0-2, vincristine 0.05 mg/kg, day 0, 7, 14) followed by three cycles of HDC (carboplatin 17 mg/kg and thiotepa 6 mg/kg, day 0 and 1) with ASCR. Histology included five medulloblastomas, four primitive neuroectodermal tumors (PNET), five malignant gliomas, and one ependymoma. Outcome and treatment toxicities were evaluated by retrospective chart review.ResultsMedian follow-up time of the 15 patients is 22 months (range 8-82 months). The 1- and 2-year progression-free survival (PFS) is 86.1% and 52.2% and overall survival (OS) 91.6% and 72.1%, respectively. Ten patients are alive and disease free 3-77 months (median 18 months) after having completed HDC/ASCR, thereoff five received XRT. Toxicity was primarily myelosuppression. There was no treatment mortality.ConclusionsWe are encouraged by the outcome of 15 children <4 yo with malignant CNS tumors treated with tandem cycles of HDC and ASCR at our institution. The treatment regimen is relatively well tolerated.(c) 2006 Wiley-Liss, Inc.

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