1. High-dose Chemotherapy With Autologous Stem Cell Rescue as First Line of Treatment in Young Children with Medulloblastoma and Supratentorial Primitive Neuroectodermal Tumors.
- Author
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Antonio Pérez-Martínez, Victor Quintero, Marta González Vicent, Julián Sevilla, Miguel Ángel Díaz, and Luis Madero
- Abstract
In order to improve the dismal prognosis of patients younger than 4 years old with medulloblastoma and supratentorial primitive neuroectodermal tumors (stPNET) seven young children were treated with high-dose chemotherapy (HDCT) and autologous stem cell rescue in our center.All patients underwent surgical debulking and standard chemotherapy. None of them received irradiation. The HDCT included busulfan 16 mg/kg, orally over 4 days (from days −5 to −2) in 6 hourly divided doses, and melphalan at a dose of 140 mg/m
2 given by intravenous infusion over 5 min on day −1. Three patients additionally received thiotepa 250 mg/m2 given by intravenous infusion daily over 2 days (from day −2 to −1) and two patients additionally received topotecan 2 mg/m2 given by intravenous infusion daily over 30 min for 5 days (from day −11 to −7).Patients' stem cells were mobilized with granulocyte colony-stimulating factor at a dose of 12 ug/kg twice daily subcutaneously for four consecutive days. Cryopreserved peripheral blood progenitor cells were reinfused 48 h after completion of chemotherapy.With a median follow-up of 21 months (range 564) five complete responses were observed; one patient had partial response and one had stable disease. There was no treatment-related mortality. The 2 year event-free survival was 71.43±17%.Therefore we conclude that HDCT as consolidation regimen may improve the cure rates in very young children with medulloblastoma/stPNET avoiding long-term sequelae of radiotherapy. [ABSTRACT FROM AUTHOR]- Published
- 2004