title: Current status of treatment for pediatric rhabdomyosarcoma in the USA and Japan.
abstract: 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.
authors.affiliation: Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto, Japan.
authors.forename: Hajime
authors.initials: H
authors.lastname: Hosoi
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D006801:Humans; D007223:Infant; D007564:Japan; D010372:Pediatrics; D018233:Rhabdomyosarcoma, Embryonal / Q000188:drug therapy* / Q000401:mortality; D018570:Risk Assessment; D015996:Survival Rate; D016896:Treatment Outcome; D014481:United States
keywords: clinical study; rhabdomyosarcoma; treatment