title: Reduced-intensity allogeneic stem cell transplantation for children with neuroblastoma who failed tandem autologous stem cell transplantation.
abstract: BACKGROUND
  To date, no effective curative option is available for children with neuroblastoma (NB) who failed tandem high-dose chemotherapy and autologous stem cell transplantation (HDCT/autoSCT). The present study evaluated the feasibility and efficacy of reduced-intensity allogeneic stem cell transplantation (RI alloSCT) in six children with NB who failed tandem HDCT/autoSCT.
  
  
  METHODS
  A cyclophosphamide/fludarabine regimen was used as a conditioning for HLA-matched SCT, and ATG was added for haploidentical SCT. Peripheral blood stem cells from four HLA-matched donors and two haploidentical donors were transplanted. Immune suppression was rapidly tapered if graft-versus-host disease (GVHD) was absent.
  
  
  RESULTS
  Regimen-related short-term toxicity was manageable, and complete donor chimerism was achieved in the early period after transplant. Grade I/II acute GVHD developed or was induced in all patients. Tumor response, attributed to a graft-versus-tumor (GVT) effect, was observed in two of six patients after induction of acute GVHD. The other four patients with significant tumor burden prior to transplant had tumor progression despite presence of GVHD. However, it was difficult to effectively reduce the tumor burden prior to transplant through the use of conventional treatment modalities.
  
  
  CONCLUSIONS
  Although regimen-related short-term toxicity was manageable in intensively pretreated patients with NB, GVT effect was not sufficiently strong to control tumor progression in patients who had a significant tumor burden at transplant. Therefore, new treatment modalities to effectively reduce tumor burden prior to transplant in concert with post-transplant adjuvant treatment to enhance the GVT effect are needed to improve the outcome after RI alloSCT.
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea. kiwoong.sung@samsung.com
authors.forename: Ki Woong
authors.initials: KW
authors.lastname: Sung
authors.forename: Jun Eun
authors.initials: JE
authors.lastname: Park
authors.forename: Hee Won
authors.initials: HW
authors.lastname: Chueh
authors.forename: Soo Hyun
authors.initials: SH
authors.lastname: Lee
authors.forename: Keon Hee
authors.initials: KH
authors.lastname: Yoo
authors.forename: Hong Hoe
authors.initials: HH
authors.lastname: Koo
authors.forename: Ju Youn
authors.initials: JY
authors.lastname: Kim
authors.forename: Eun Joo
authors.initials: EJ
authors.lastname: Cho
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D002675:Child, Preschool; D005260:Female; D020350:Graft vs Tumor Effect; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D009447:Neuroblastoma / Q000401:mortality / Q000601:surgery*; D016879:Salvage Therapy / Q000009:adverse effects / Q000379:methods*; D016019:Survival Analysis; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous / Q000009:adverse effects / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents