title: Tandem stem cell rescue as consolidation therapy for high-risk neuroblastoma.
abstract: BACKGROUND
  Despite aggressive treatment for high-risk neuroblastoma (NB), event-free survival (EFS) remains <40%. In single arm studies, intensifying therapy with high-dose chemotherapy and tandem autologous stem cell rescue (HDC/SCR) improved outcome. We retrospectively describe our institutional experience in using HDC/SCR for patients with high-risk NB, focusing on outcome and acute toxicities.
  
  
  METHODS
  Eighty-four patients with high-risk NB at Children's Healthcare of Atlanta treated over a 12-year time period underwent HDC/SCR as part of upfront therapy; 28 patients received a single HDC/SCR and 56 patients received tandem HDC/SCR. The two groups were compared in terms of EFS, overall survival (OS), and acute transplant related toxicities.
  
  
  RESULTS
  Patients who received tandem HDC/SCR had a significantly improved EFS compared with patients who received a single HDC/SCR (4-year EFS 59.3 ± 6.7% vs. 26.8 ± 9.2%, P=0.01). Similarly, the 4-year OS was improved in patients receiving tandem HDC/SCR, though this did not reach statistical significance (70.6 ± 9.2% vs. 44.7±11.2%, P=0.06). Multivariate regression confirmed the prognostic role of the treatment group. None of the patients who underwent a single HDC/SCR developed veno-occlusive disease (VOD), while 17% of patients who underwent tandem HDC/SCR developed mild-to-severe VOD. Rates of other transplant-related acute toxicities were similar.
  
  
  CONCLUSIONS
  Tandem HDC/SCR for patients with high-risk NB seems to improve survival without significant increases in acute toxicities. This needs to be validated in randomized prospective trials.
authors.affiliation: Aflac Cancer Center, Division of Pediatric Hematology/Oncology, Emory University and Children's Healthcare of Atlanta, GA 30322, USA.
authors.forename: Muna
authors.initials: M
authors.lastname: Qayed
authors.forename: Kuang-Yueh
authors.initials: KY
authors.lastname: Chiang
authors.forename: Richard
authors.initials: R
authors.lastname: Ricketts
authors.forename: Adina
authors.initials: A
authors.lastname: Alazraki
authors.forename: Ali
authors.initials: A
authors.lastname: Tahvildari
authors.forename: Ann
authors.initials: A
authors.lastname: Haight
authors.forename: Brad
authors.initials: B
authors.lastname: George
authors.forename: Natia
authors.initials: N
authors.lastname: Esiashvili
authors.forename: Howard M
authors.initials: HM
authors.lastname: Katzenstein
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D006801:Humans; D060828:Induction Chemotherapy / Q000009:adverse effects; D007223:Infant; D008297:Male; D015999:Multivariate Analysis; D009447:Neuroblastoma / Q000628:therapy*; D012189:Retrospective Studies; D033581:Stem Cell Transplantation / Q000009:adverse effects; D016019:Survival Analysis; D055815:Young Adult