title: A pilot trial of tandem autologous peripheral blood progenitor cell transplantation following high-dose thiotepa and carboplatin in children with poor-risk central nervous system tumors.
abstract: This is a pilot study performed to determine the maximum tolerated number of courses of high-dose thiotepa and carboplatin with autologous peripheral blood progenitor cell (PBPC) transplantation in poor-risk pediatric central nervous system (CNS) tumor patients. Twelve patients were enrolled and a total of 24 PBPC transplants were performed. The median age was 7.7 years. All patients had CNS tumors: 4 relapsed CNS PNET, 2 high-risk PNET in first remission, 2 relapsed/progressive brainstem tumor, 2 relapsed/progressive anaplastic astrocytoma, 1 relapsed GBM, and 1 recurrent ependymoma. The regimen consisted of thiotepa 250 mg/m2/day x 3 days and carboplatin 400 mg/m2/day x 3 days. No toxic deaths occurred. All patients were hospitalized for a median duration of 17 days. The median number of CD34 cells infused was 5.4 x 10(6)/kg (2.1-29.7 x 10(6)/kg) per course. Median time to ANC > 0.5 x 10(9)/L was 9 days, and platelets > 20 x 10(9)/L was 13.5 days. Four patients came off protocol after only one course of PBPC (2 had tumor progression, 2 parental choice); 4 patients underwent two, and 4 patients three courses of PBPC. Major nonhematologic complications were mucositis that necessitated infusion of narcotics (11/24 courses), fever of unknown origin (12/24), documented infection (9/24), and hemorrhagic cystitis (3/24). TPN was administered during 22 of 24 courses with a median duration of 15 days. It isfeasible to administer 2-3 courses of tandem high-dose thiotepa and carboplatin with PBPC transplant with prompt engraftment and manageable toxicities in pediatric CNS tumor patients.
authors.affiliation: New York Medical College, Department of Pediatrics, Section of Hematology/Oncology and Blood and Marrow Transplantation, Valhalla, NY 10595, USA. mehmet_ozkaynak@nymc.edu
authors.forename: M Fevzi
authors.initials: MF
authors.lastname: Ozkaynak
authors.forename: Claudio
authors.initials: C
authors.lastname: Sandoval
authors.forename: Oya
authors.initials: O
authors.lastname: Levendoglu-Tugal
authors.forename: Somasundaram
authors.initials: S
authors.lastname: Jayabose
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D016190:Carboplatin / Q000008:administration & dosage; D016543:Central Nervous System Neoplasms / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D033581:Stem Cell Transplantation; D016019:Survival Analysis; D013852:Thiotepa / Q000008:administration & dosage; D014182:Transplantation, Autologous
chemical_list: D013852:Thiotepa; D016190:Carboplatin