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Current status of treatment for pediatric rhabdomyosarcoma in the USA and Japan
Author(s) -
Hosoi Hajime
Publication year - 2016
Publication title -
pediatrics international
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.49
H-Index - 63
eISSN - 1442-200X
pISSN - 1328-8067
DOI - 10.1111/ped.12867
Subject(s) - medicine , rhabdomyosarcoma , vincristine , regimen , cyclophosphamide , irinotecan , topotecan , chemotherapy , surgery , gastroenterology , oncology , cancer , sarcoma , pathology , colorectal cancer
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.

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