Abstract
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 language | English |
|---|---|
| Pages (from-to) | 3035-3041 |
| Number of pages | 7 |
| Journal | European Journal of Cancer |
| Volume | 45 |
| Issue number | 17 |
| DOIs | |
| Publication status | Published - Nov 2009 |
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Keywords
- High-dose chemotherapy
- Metastases
- Rhabdomyosarcoma
- Soft tissue sarcoma
ASJC Scopus subject areas
- Cancer Research
- Oncology
Cite this
Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma. / Bisogno, Gianni; Ferrari, Andrea; Prete, Arcangelo; Messina, Chiara; Basso, Eleonora; Cecchetto, Giovanni; Indolfi, Paolo; Scarzello, Giovanni; D'Angelo, Paolo; Sio, Luigi De; Di Cataldo, Andrea; Carli, Modesto.
In: European Journal of Cancer, Vol. 45, No. 17, 11.2009, p. 3035-3041.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma
AU - Bisogno, Gianni
AU - Ferrari, Andrea
AU - Prete, Arcangelo
AU - Messina, Chiara
AU - Basso, Eleonora
AU - Cecchetto, Giovanni
AU - Indolfi, Paolo
AU - Scarzello, Giovanni
AU - D'Angelo, Paolo
AU - Sio, Luigi De
AU - Di Cataldo, Andrea
AU - Carli, Modesto
PY - 2009/11
Y1 - 2009/11
N2 - 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.
AB - 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.
KW - High-dose chemotherapy
KW - Metastases
KW - Rhabdomyosarcoma
KW - Soft tissue sarcoma
UR - http://www.scopus.com/inward/record.url?scp=70350602712&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=70350602712&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2009.08.019
DO - 10.1016/j.ejca.2009.08.019
M3 - Article
VL - 45
SP - 3035
EP - 3041
JO - European Journal of Cancer
JF - European Journal of Cancer
SN - 0959-8049
IS - 17
ER -