Aim: The RMS4.99 study was designed to explore the role of multiple sequential high-dosechemotherapy cycles administered early in the treatment of children with metastatic rhabdomyosarcoma.Patients and methods: Seventy patients were enrolled and received three cycles of initialstandard chemotherapy, followed by a course of cyclophosphamide and etoposide to obtainperipheral blood stem cells (PBSC), then three consecutive high-dose combinations followedby PBSC rescue. This was followed by surgery and/or radiotherapy, after which a finalmaintenance treatment with six courses of vincristine, actinomycin D and cyclophosphamidewas administered.Results: Sixty-two patients underwent the high-dose chemotherapy phase. The 3-yearoverall survival (OS) and progression free survival (PFS) rates for the 70 patients were42.3% (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 ofpatients with only one or no unfavourable prognostic factors (age > 10 years, unfavourablesite 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 characteristicshave a better survival. The use of sequential cycles of high-dose chemotherapy did notappear of benefit for patients with metastatic rhabdomyosarcoma.

Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma

DI CATALDO, Andrea;
2009-01-01

Abstract

Aim: The RMS4.99 study was designed to explore the role of multiple sequential high-dosechemotherapy cycles administered early in the treatment of children with metastatic rhabdomyosarcoma.Patients and methods: Seventy patients were enrolled and received three cycles of initialstandard chemotherapy, followed by a course of cyclophosphamide and etoposide to obtainperipheral blood stem cells (PBSC), then three consecutive high-dose combinations followedby PBSC rescue. This was followed by surgery and/or radiotherapy, after which a finalmaintenance treatment with six courses of vincristine, actinomycin D and cyclophosphamidewas administered.Results: Sixty-two patients underwent the high-dose chemotherapy phase. The 3-yearoverall survival (OS) and progression free survival (PFS) rates for the 70 patients were42.3% (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 ofpatients with only one or no unfavourable prognostic factors (age > 10 years, unfavourablesite 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 characteristicshave a better survival. The use of sequential cycles of high-dose chemotherapy did notappear of benefit for patients with metastatic rhabdomyosarcoma.
2009
Rhabdomyosarcoma; Metastases; Soft tissue sarcoma; High-dose chemotherapy
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11769/6940
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