Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma

Gianni Bisogno, Andrea Ferrari, Arcangelo Prete, Chiara Messina, Eleonora Basso, Giovanni Cecchetto, Paolo Indolfi, Giovanni Scarzello, Paolo D'Angelo, Luigi De Sio, Andrea Di Cataldo, Modesto Carli

Research output: Contribution to journalArticlepeer-review


Aim: The RMS4.99 study was designed to explore the role of multiple sequential high-dose chemotherapy cycles administered early in the treatment of children with metastatic rhabdomyosarcoma. Patients and methods: Seventy patients were enrolled and received three cycles of initial standard chemotherapy, followed by a course of cyclophosphamide and etoposide to obtain peripheral blood stem cells (PBSC), then three consecutive high-dose combinations followed by PBSC rescue. This was followed by surgery and/or radiotherapy, after which a final maintenance treatment with six courses of vincristine, actinomycin D and cyclophosphamide was administered. Results: Sixty-two patients underwent the high-dose chemotherapy phase. The 3-year overall survival (OS) and progression free survival (PFS) rates for the 70 patients were 42.3% (95% confidence interval [CI] 39.5-53.6) and 35.3% (95% CI, 24.3-46.5), respectively. By multivariate analysis survival correlated strongly with age > 10 years. In a subset of patients with only one or no unfavourable prognostic factors (age > 10 years, unfavourable site of primary tumour, bone or bone marrow involvement and number of metastatic sites >2) the PFS was significantly higher, i.e. 60.5% at 3 years. Conclusion: Our study confirms that patients with favourable prognostic characteristics have a better survival. The use of sequential cycles of high-dose chemotherapy did not appear of benefit for patients with metastatic rhabdomyosarcoma.

Original languageEnglish
Pages (from-to)3035-3041
Number of pages7
JournalEuropean Journal of Cancer
Issue number17
Publication statusPublished - Nov 2009


  • High-dose chemotherapy
  • Metastases
  • Rhabdomyosarcoma
  • Soft tissue sarcoma

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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