title: Adjuvant chemotherapy for the treatment of advanced pediatric nonrhabdomyosarcoma soft tissue sarcoma: the National Cancer Institute experience.
abstract: BACKGROUND
  The survival of children and adolescents with advanced (unresectable or metastatic) nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) is poor. In order to clarify the role of combining chemotherapy with aggressive local control using surgery and/or radiation, we reviewed our institutional experience with the treatment of advanced pediatric NRSTS.
  
  
  METHODS
  We reviewed the charts of all patients less than 21 years treated for an advanced NRSTS at the National Cancer Institute (NCI) between 1983 and 2003. Tumor pathology was confirmed and demographic, disease, and treatment data were abstracted. Survival was calculated using standard methods.
  
  
  RESULTS
  Of the 25 patients who were treated over the study period, 15 had metastatic disease and 10 had unresectable or incompletely resected disease at presentation. Twenty-one patients received chemotherapy consisting of the combination of vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide, and the remaining 4 received regimens that included doxorubicin. Twenty patients (80%) had a complete (5/25) or partial (15/25) response after chemotherapy alone. After the combination of chemotherapy and local control, 14 patients (56%) had a complete response (CR). The estimated 5-year overall and event-free survival (EFS) for all patients was 0.50 (standard error = 0.11) and 0.34 (standard error = 0.10), respectively.
  
  
  CONCLUSIONS
  The combination of chemotherapy with aggressive local control in this cohort of pediatric patients with advanced NRSTS yielded results comparable to those observed in patients with advanced sarcomas that are chemotherapy responsive. Prospective randomized trials are needed to quantify the contribution of chemotherapy and to determine the ideal regimen.
authors.affiliation: National Cancer Institute, Pediatric Oncology Branch, Bethesda, MD 20892, USA.
authors.forename: Paul C
authors.initials: PC
authors.lastname: Nathan
authors.forename: Maria
authors.initials: M
authors.lastname: Tsokos
authors.forename: Lauren
authors.initials: L
authors.lastname: Long
authors.forename: Donna
authors.initials: D
authors.lastname: Bernstein
authors.forename: Leonard H
authors.initials: LH
authors.lastname: Wexler
authors.forename: Crystal L
authors.initials: CL
authors.lastname: Mackall
authors.forename: Lee J
authors.initials: LJ
authors.lastname: Helman
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D004317:Doxorubicin / Q000627:therapeutic use; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009362:Neoplasm Metastasis; D012074:Remission Induction / Q000379:methods; D012189:Retrospective Studies; D012983:Soft Tissue Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D016019:Survival Analysis
chemical_list: D004317:Doxorubicin