Role of high-dose chemotherapy with hematopoietic stem cell rescue in the treatment of metastatic or recurrent rhabdomyosarcoma

J Pediatr Hematol Oncol. Jun-Jul 2001;23(5):272-6. doi: 10.1097/00043426-200106000-00007.


Purpose: 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).

Patients and 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.

Publication types

  • Meta-Analysis

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Bone Marrow Diseases / chemically induced
  • Bone Marrow Diseases / therapy
  • Child
  • Child, Preschool
  • Clinical Trials as Topic / methods
  • Combined Modality Therapy
  • Cyclophosphamide / administration & dosage
  • Disease-Free Survival
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Life Tables
  • Neoplasm Metastasis
  • Neoplasm Recurrence, Local
  • Neoplasm Staging
  • Prospective Studies
  • Remission Induction
  • Rhabdomyosarcoma / drug therapy*
  • Rhabdomyosarcoma / mortality
  • Rhabdomyosarcoma / pathology
  • Rhabdomyosarcoma / surgery
  • Risk
  • Soft Tissue Neoplasms / drug therapy*
  • Soft Tissue Neoplasms / mortality
  • Soft Tissue Neoplasms / pathology
  • Soft Tissue Neoplasms / surgery
  • Survival Analysis
  • Thiotepa / administration & dosage
  • Transplantation Conditioning
  • Treatment Outcome
  • Whole-Body Irradiation


  • Cyclophosphamide
  • Thiotepa