• 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, and Modesto Carli.
• Division of Haematology/Oncology, Department of Paediatrics, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy. gianni.bisogno@unipd.it
• Eur. J. Cancer. 2009 Nov 1; 45 (17): 3035-41.

AimThe 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 MethodsSeventy 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.ResultsSixty-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.ConclusionOur 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.

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