title: Does consolidation with autologous stem cell transplantation improve the outcome of children with metastatic or relapsed Ewing sarcoma?
abstract: OBJECTIVE
  To evaluate the role of high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) as consolidation therapy for children with high-risk Ewing sarcoma (ES) treated at The Hospital for Sick Children (SickKids), Toronto.
  
  
  METHODS
  The charts of children treated for high-risk ES (defined as metastatic at diagnosis or relapsed) between 1990 and 2005 at SickKids were reviewed. Forty-five children were identified. Twenty patients received ASCT after induction with vincristine, doxorubicin, ifosfamide, cyclophosphamide, and etoposide. Patients with resectable tumor or lung metastases underwent surgery and those with non-resectable tumors were treated with irradiation. Twenty-five patients were treated with conventional chemotherapy (CC). Primary metastatic patients were treated with either a local protocol or as per POG 9354. At relapse, patients were treated with topotecan, cyclophosphamide, then ifosfamide, carboplatin, and etoposide (ICE). Local control was attained with surgery and/or irradiation.
  
  
  RESULTS
  Ten of the 20 patients treated with ASCT are alive (median follow-up 6 years), with 8/10 being in remission more than 5 years from diagnosis. The 3-year overall survival (OS) for ASCT was 59%, (95% CI: 36%, 81%) compared to 34% (14%, 53%) for patients treated with CC (P-value = 0.06). The 3-year event-free survival (EFS) for the ASCT was 39% (17%, 60%) compared to 32% (13%, 50%) in the CC group (P = 0.08).
  
  
  CONCLUSIONS
  ASCT appears to add some benefit to conventional multimodality therapy for children with high-risk ES. Randomized controlled trials are warranted.
authors.affiliation: Department of Pediatrics, Division of Hematology, The Hospital for Sick Children, University of Toronto, Ontario, Canada. nafisaha@gmail.com
authors.forename: Nafisah
authors.initials: N
authors.lastname: Al-Faris
authors.forename: Talal
authors.initials: T
authors.lastname: Al Harbi
authors.forename: Cristina
authors.initials: C
authors.lastname: Goia
authors.forename: Alberto
authors.initials: A
authors.lastname: Pappo
authors.forename: John
authors.initials: J
authors.lastname: Doyle
authors.forename: Adam
authors.initials: A
authors.lastname: Gassas
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000627:therapeutic use*; D001859:Bone Neoplasms / Q000473:pathology; D002166:Camptothecin / Q000008:administration & dosage / Q000031:analogs & derivatives; D016190:Carboplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D004317:Doxorubicin / Q000008:administration & dosage; D015251:Epirubicin / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005500:Follow-Up Studies; D006801:Humans; D007069:Ifosfamide / Q000008:administration & dosage; D017053:Infection Control; D053208:Kaplan-Meier Estimate; D008558:Melphalan / Q000008:administration & dosage; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy / Q000532:radiotherapy / Q000601:surgery*; D036102:Peripheral Blood Stem Cell Transplantation / Q000706:statistics & numerical data*; D011182:Postoperative Care / Q000379:methods; D012189:Retrospective Studies; D012306:Risk; D016879:Salvage Therapy; D012512:Sarcoma, Ewing / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000556:secondary* / Q000601:surgery*; D019772:Topotecan / Q000008:administration & dosage; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D017211:Treatment Failure; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D015251:Epirubicin; D014750:Vincristine; D005047:Etoposide; D019772:Topotecan; D004317:Doxorubicin; D003520:Cyclophosphamide; D016190:Carboplatin; C095887:exatecan; D008558:Melphalan; D007069:Ifosfamide; D002166:Camptothecin