title: High-dose chemotherapy with autologous stem cell rescue as first line of treatment in young children with medulloblastoma and supratentorial primitive neuroectodermal tumors.
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/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.
authors.affiliation: Department of Pediatric Hematology and Oncology, Hospital Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain.
authors.forename: Antonio
authors.initials: A
authors.lastname: Pérez-Martínez
authors.forename: Victor
authors.initials: V
authors.lastname: Quintero
authors.forename: Marta González
authors.initials: MG
authors.lastname: Vicent
authors.forename: Julián
authors.initials: J
authors.lastname: Sevilla
authors.forename: Miguel Angel
authors.initials: MA
authors.lastname: Díaz
authors.forename: Luis
authors.initials: L
authors.lastname: Madero
mesh_terms: D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage / Q000009:adverse effects; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D002066:Busulfan / Q000008:administration & dosage / Q000009:adverse effects; D002528:Cerebellar Neoplasms / Q000401:mortality / Q000628:therapy*; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008527:Medulloblastoma / Q000401:mortality / Q000628:therapy*; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D018242:Neuroectodermal Tumors, Primitive / Q000401:mortality / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000009:adverse effects; D015173:Supratentorial Neoplasms / Q000401:mortality / Q000628:therapy*; D013852:Thiotepa / Q000008:administration & dosage / Q000009:adverse effects; D019772:Topotecan / Q000008:administration & dosage / Q000009:adverse effects; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D019772:Topotecan; D013852:Thiotepa; D002066:Busulfan; D008558:Melphalan