Treatment > Busulfan · Melphalan · Stem Cells · Thiotepa · Topotecan

Abstract

Antonio Pérez-Martínez, Alvaro Lassaletta, Marta González-Vicent, Julián Sevilla, Miguel Angel Díaz and Luis Madero

Department of Pediatric Hematology and Oncology, Hospital Niño Jesús, Universidad Autónoma de Madrid, Spain [A.P.-M., A.L., M.G.-V., J.S., M.A.D., L.M.]. Department of Pediatric Hematology–Oncology, Hospital Niño Jesús, Avda, Menendez Pelayo 65, 28009 Madrid, Spain [L.M.].
Correspondence to: Luis Madero, Email: lmadero.hnjs@salud.madrid.org, Phone: +34-91-5035938, Fax: +34-91-5035902.

Current treatment for high risk and recurrent medulloblastoma (MB) and supratentorial primitive neuroectodermal tumors (stPNET) has a very poor prognosis in children. 
High dose chemotherapy (HDCT) and autologous stem cell rescue have improved survival rates. 
We present 19 patients (thirteen classified in the high risk group and six patients with recurrent disease) that received HDCT and autologous stem cell rescue. 
In the high risk group [Med Pediatr Oncol 38 (2002) 83], all patients underwent neurosurgical debulking. 
Standard chemotherapy was prescribed in 10 patients. 
Radiotherapy was given to 4 patients (all older than 4 years old). 
In the recurrence disease group [Childs Nerv Syst 15 (1999) 498], five patients underwent surgery. 
Radiotherapy was given to those who were not previously irradiated. 
The HDCT in twelve patients consisted of busulfan 4 mg/kg/day, orally over 4 days in 6-hourly divided doses and melphalan at a dose of 140 mg/m²/day by intravenous infusion over 5 min on day –1. 
Three patients additionally received thiotepa 250 mg/m²/day intravenously over 2 days and four patients additionally received topotecan 2 mg/m²/day over 5 days by intravenous infusion over 30min. 
The other seven patients received busulfan and thiotepa at the same doses. 
Patient's stem cells were mobilized with granulocyte colony-stimulating factor at a dose of 12 μg/kg twice daily subcutaneously for four consecutive days. 
Cryopreserved peripheral blood progenitor cells were re-infused 48h after completion of chemotherapy. 
With a median follow-up of 34 months (range 5–93) eight complete responses and one partial response were observed. 
Three patients died of treatment-related toxicities (15%). 
The 2 year event-free survival was 37.67 ± 14% in all patients and 57 ± 15% for the high risk group. 
Therefore we conclude that HDCT may improve survival rates in patients with high risk/recurrent MB and stPNET despite treatment toxicity.

Keywords: autologous peripheral blood progenitor cell transplantation - high-dose chemotherapy - high-risk brain tumors - medulloblastoma - supratentorial primitive neuroectodermal tumors

© Springer 2005

Source: http://springerlink.metapress.com/openurl.asp?genre=article&eissn=1573-7373&volume=71&issue=1&spage=33
DOI: http://dx.doi.org/10.1007/s11060-004-4527-4