Regardless of improvement in cure of Rhabdomyosarcoma (RMS), the results in treatment of advanced stage of RMS in children are still dismal. Recently, high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation (HDC/APBSCT) has been tried to manage the advanced high-risk RMS patients. We investigated the effectiveness of HDC/APBSCT by reviewing the clinical records of high-risk pediatric RMS patients in single institute database. Over twenty years, 37 patients were diagnosed as RMS with high-risk at the time of first diagnosis. These patients were classified as two groups according to treatment method. The first group was HDC/APBSCT and the other was conventional multi-agent chemotherapy group. Differences of clinical results between the two groups were analyzed. The median age of patients was 5 yr, ranging from 6 months to 15 yr. The 5-yr event free survival rate (EFS) of all patients was 24.8% +/- 4.8%. HDC/APBSCT group and conventional multi-agent chemotherapy group were 41.3% +/- 17.8% and 16.7% +/- 7.6% for 5-yr EFS, respectively (P = 0.023). There was a significant difference in the result of HDC/APBSCT between complete remission or very good partial response group and poor response group (50% +/- 20.4% vs 37.5% +/- 28.6%, P = 0.018). HDC/APBSCT can be a promising treatment modality in high-risk RMS patients.
Adolescent ; Antineoplastic Combined Chemotherapy Protocols/*therapeutic use ; Carboplatin/administration & dosage ; Child ; Child, Preschool ; Disease-Free Survival ; Etoposide/administration & dosage ; Female ; Humans ; Ifosfamide/administration & dosage ; Infant ; Male ; *Peripheral Blood Stem Cell Transplantation ; Retrospective Studies ; Rhabdomyosarcoma/*drug therapy/mortality/pathology ; Survival Rate ; Transplantation, Autologous ; Treatment Outcome