Background: In the last few years, survival in children with central nervous system (CNS) tumors has slightly improved, especially in children with tumors such as medulloblastoma and those in which complete surgical resection is achieved. However, outcome remains poor in patients with incomplete surgical resection, neuroaxial dissemination, metastatic or recurrent tumors and in very young children.
Objectives: To improve prognosis in patients with high-risk and recurrent tumors, new therapeutic strategies such as high-dose chemotherapy with autologous stem cell rescue (ASCR) have been developed.
Methods: We retrospectively studied patients with high-risk and recurrent CNS tumors who underwent ASCR between September 1995 and December 2002 in our unit.
Results: Thirty-five patients underwent ASCR. Seven patients died of treatment-related toxicities (20 %). Thirteen (37 %) are event-free survivors at a median post-ASCR follow-up of 18 months (range: 5-63 months). The 2-year Kaplan-Meier estimates of event-free survival was 37.64 +/- 8.7 % in all patients, 57 +/- 15 % in the group of patients with high-risk medulloblastoma/supratentorial primitive neuroectodermal tumor (stPNET) and 71.43 +/- 17 % in patients aged less than 4 years with medulloblastoma/stPNET.
Conclusions: In our experience, ASCR may be effective in the treatment of malignant tumors of the central nervous system in patients with controlled disease, in certain histologic groups and chemosensitive tumors (medulloblastoma, malignant astrocytoma), as well as in very young children in whom cranial radiotherapy is contraindicated.