Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma.
OBJECTIVE
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.


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.


CONCLUSIONS
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.
Division of Haematology/Oncology, Department of Paediatrics, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy. gianni.bisogno@unipd.it
Gianni
G
Bisogno
Andrea
A
Ferrari
Arcangelo
A
Prete
Chiara
C
Messina
Eleonora
E
Basso
Giovanni
G
Cecchetto
Paolo
P
Indolfi
Giovanni
G
Scarzello
Paolo
P
D'Angelo
Luigi
L
De Sio
Andrea
A
Di Cataldo
Modesto
M
Carli
D000293:Adolescent; D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D004334:Drug Administration Schedule; D004812:Epidemiologic Methods; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation; D011379:Prognosis; D012208:Rhabdomyosarcoma / Q000188:drug therapy* / Q000473:pathology / Q000556:secondary*; D016896:Treatment Outcome; D055815:Young Adult