This article reviews the current status of treatment for children with
rhabdomyosarcoma, according to the four risk groups. Low-risk subgroup A: the Children's Oncology Group in the USA recently performed a clinical trial consisting of a
chemotherapy regimen with a shortened treatment period and a reduced
drug dosage. Patients in this group received only four cycles of
vincristine and
actinomycin D (VA) after four cycles of
vincristine,
actinomycin D, and
cyclophosphamide (VAC) with
cyclophosphamide (CPM) 1.2 g/m(2) and their outcome was no worse than that obtained with previous regimens. Low-risk subgroup B: although marked improvement in survival was seen with an intensive
VAC regimen with CPM 2.2 g/m(2) /cycle (Intergroup
Rhabdomyosarcoma Study [IRS]-V, 1997-2004), the total dose of CPM in this regimen caused serious and fatal
hepatic veno-occlusive disease during treatment and probably cannot avoid
infertility or possible secondary
cancer as a late effect. Thereafter, a reduced-dose regimen consisting of four cycles of VAC with CPM 1.2 g/m(2) followed by 12 cycles of VA was investigated in the next study, but the outcome appeared to be worse than in IRS-V. Intermediate-risk group: no significant difference was found between VAC/
vincristine,
topotecan and cyclophispahamide (VTC) and intensive VAC in IRS-V. The results of a subsequent regimen of VAC with CPM 1.2 g/m(2) alternating with
vincristine and
irinotecan are awaited. High-risk group: overall survival is approximately 30% and has not improved over the last 25 years. Although 18 month failure-free survival (FFS) was improved with an intensive combination
therapy regimen, 36 month FFS dropped to 32% and thus better novel approaches or additive treatments are needed.