Clinical results of high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation in children with advanced stage rhabdomyosarcoma

Abstract

Purpose: Rhabdomyosarcoma (RMS) is most common soft-tissue sarcoma and highly malignant tumor in children. Regardless of improvement in cure of RMS, the results in treatment of advanced stage of RMS 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 RMS patients in single institute data base.Methods: Over twenty years, 37 patients were diagnosed as RMS with high-risk at the time of first diagnosis in Severance Hospital, Seoul, Korea. High-risk patients were defined as clinical group III or IV. 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 data of patients were reviewed retrospectively.Result: Seventeen and twenty patients were female and male, respectively. The median age of patients was 5 years, ranging from 6 months to 15 years. The 5-year overall survival rate (OS) of all patients was about 25.1±7.6%. HDC/APBSCT group and conventional multi-agent chemotherapy group were 40.5±16.5% and 16.7±7.6% for 5-year OS, respectively (p=0.028). There was a significant difference in the result of HDC/APBSCT between complete remission or very good partial response group and poor response group. The difference of OS between two groups was 51.4±20.4% versus 25±21.7% (p=0.04). Especially, in the very high-risk group (Group IV or Stage IV over 10 years of age with embryonal histology or all alveolar histology), there was statistically significant different OS between HDC/APBSCT and conventional multi-agent chemotherapy group (32.7±17.3% vs. 0%).Conclusion: HDC/APBSCT can be a promising treatment modality, especially in very high-risk RMS patients. It should be performed to patients with good response to conventional multi-agent chemotherapy because good outcome and tolerable treatment related toxicity are expected.