title: Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: a Pediatric Oncology Group study: a report from the Children's Oncology Group.
abstract: BACKGROUND
  The Pediatric Oncology Group performed a pilot study to assess the feasibility of tandem high dose chemotherapy (HDC) with stem cell rescue (HDC/SCR). We report here the results of this single arm trial of induction chemotherapy, local control measures (surgery and local radiation), and tandem HDC/SCR.
  
  
  METHODS
  Patients with high risk neuroblastoma (NBL) underwent five cycles of induction chemotherapy and resection of primary tumors. Peripheral blood stem cells (PBSC) were collected after Course 3 without exvivo manipulation. Myeloablative chemotherapy was performed in rapid sequence after induction chemotherapy and surgery. The ability of patients to complete both cycles of HDC/SCR was a primary endpoint. Transplant-related toxicity, progression-free survival (PFS) and overall survival (OS) were recorded.
  
  
  RESULTS
  A total of 33 patients were enrolled. Twenty-two patients completed at least one HDC/SCR procedure and 17 patients completed both. Only one patient had insufficient stem cells collected for both transplants. There was one transplant-related death; engraftment was rapid and toxicity was as expected. The PFS of the 33 patients treated on this study is 24.2% ± 7.5% and OS is 36.4% ± 8.4% at 5 years. For patients who received at least one transplant PFS is 36.4% ± 11.0% and OS is 45.5% ± 11.2% at 5 years.
  
  
  CONCLUSIONS
  The treatment of high risk NBL with tandem HDC/SCR is feasible in terms of transplant-related mortality and the ability to collect adequate PBSC for two transplants. The outcomes from this intensified treatment have been used to design a Children's Oncology Group Phase III study testing the efficacy of tandem HDC/SCR.
authors: Department of Hematology and Oncology, Cook Children's Medical Center, Fort Worth, Texas 76104, USA. meaghan.grangermd@cookchildrens.org
authors: Meaghan
authors: M
authors: Granger
authors: Stephan A
authors: SA
authors: Grupp
authors: Morris
authors: M
authors: Kletzel
authors: Cynthia
authors: C
authors: Kretschmar
authors: Arlene
authors: A
authors: Naranjo
authors: Wendy B
authors: WB
authors: London
authors: Lisa
authors: L
authors: Diller
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D006801:Humans; D007223:Infant; D008297:Male; D009447:Neuroblastoma / Q000401:mortality* / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D015996:Survival Rate; D014182:Transplantation, Autologous