title: Role of high-dose chemotherapy with hematopoietic stem cell rescue in the treatment of metastatic or recurrent rhabdomyosarcoma.
abstract: OBJECTIVE
  This review summarizes the published data on the use of high-dose chemotherapy and hematopoietic stem cell rescue (HSCR) in the treatment of recurrent or metastatic rhabdomyosarcoma (RMS).
  
  
  METHODS
  Three hundred eighty-nine patients were identified from 22 articles selected by computer generated searching of MEDLINE (1979-present). One hundred seventy-seven patients had stage 4 disease and were treated during first complete remission (CRI). The remaining patients were treated during CR1/first partial remission (PR1) (110 patients), CR2/PR2 (53 patients), CR2 (12 patients), CR3 (1 patient), or treated with disease (36 patients).
  
  
  RESULTS
  Patients treated during CR1 or CR1/PR1 had event-free survival (EFS) rates ranging from 24% to 29% at 3 to 6 years from diagnosis and overall survival (OS) rates ranging from 20% to 40% at 2 to 6 years after diagnosis according to data provided as Kaplan-Meier estimates. Studies without Kaplan-Meier estimates (n = 32) indicate that 12 patients (38%) with stage IV RMS treated during CR1 or CR1/PR1 were surviving 7 to 60 months from diagnosis, similar to patients with stage IV RMS treated on Intergroup Rhabdomyosarcoma Studies II or III. Patients treated during CR2, CR3, or with evidence of disease had a worse outcome with an estimated 3 years OS of 12% (n = 51). Studies without Kaplan-Meier estimates (n = 27) indicate that four patients (15%) treated during CR2, CR3, or with disease were surviving 17 to 33 months after transplant.
  
  
  CONCLUSIONS
  Based on these data, there does not appear to be a significant advantage to undergoing high-dose chemotherapy with HSCR for patients with relapsed or refractory high-risk RMS. Clearly, there is a need for incorporating new treatment strategies for patients with high-risk RMS.
authors.affiliation: Department of Pediatrics, University of Minnesota, Minneapolis 55455, USA. weige007@tc.umn.edu
authors.forename: B J
authors.initials: BJ
authors.lastname: Weigel
authors.forename: P P
authors.initials: PP
authors.lastname: Breitfeld
authors.forename: D
authors.initials: D
authors.lastname: Hawkins
authors.forename: W M
authors.initials: WM
authors.lastname: Crist
authors.forename: K S
authors.initials: KS
authors.lastname: Baker
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001855:Bone Marrow Diseases / Q000139:chemically induced / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000379:methods; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D015993:Life Tables; D016239:MEDLINE; D009362:Neoplasm Metastasis; D009364:Neoplasm Recurrence, Local; D009367:Neoplasm Staging; D011446:Prospective Studies; D012074:Remission Induction; D012208:Rhabdomyosarcoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology / Q000601:surgery; D012306:Risk; D012983:Soft Tissue Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology / Q000601:surgery; D016019:Survival Analysis; D013852:Thiotepa / Q000008:administration & dosage; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014916:Whole-Body Irradiation
chemical_list: D003520:Cyclophosphamide; D013852:Thiotepa