Treatment > Busulfan · Stem Cells  

Abstract

Department of Pediatric Hematology and Oncology, Hospital Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain

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/m2 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 microg/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 5-64) 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.

PMID: 15072454 [PubMed]

Source: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=15072454