High-Dose Chemotherapy Followed by Autologous Stem Cell Transplantation for Metastatic Rhabdomyosarcoma—A Systematic Review

INTRODUCTION: Patients with metastatic rhabdomyosarcoma (RMS) have a poor prognosis. The aim of this systematic review is to investigate whether high-dose chemotherapy (HDCT) followed by autologous hematopoietic stem cell transplantation (HSCT) in patients with metastatic RMS has additional benefit or harm compared to standard chemotherapy. METHODS: Systematic literature searches were performed in MEDLINE, EMBASE, and The Cochrane Library. All databases were searched from inception to February 2010. PubMed was searched in June 2010 for a last update. In addition to randomized and non-randomized controlled trials, case series and case reports were included to complement results from scant data. The primary outcome was overall survival. A meta-analysis was performed using the hazard ratio as primary effect measure, which was estimated from Cox proportional hazard models or from summary statistics of Kaplan Meier product-limit estimations. RESULTS: A total of 40 studies with 287 transplant patients with metastatic RMS (age range 0 to 32 years) were included in the assessment. We identified 3 non-randomized controlled trials. The 3-year overall survival ranged from 22% to 53% in the transplant groups vs. 18% to 55% in the control groups. Meta-analysis on overall survival in controlled trials showed no difference between treatments. Result of meta-analysis of pooled individual survival data of case series and case reports, and results from uncontrolled studies with aggregate data were in the range of those from controlled data. The risk of bias was high in all studies due to methodological flaws. CONCLUSIONS: HDCT followed by autologous HSCT in patients with RMS remains an experimental treatment. At present, it does not appear justifiable to use this treatment except in appropriately designed controlled trials

The significance of margins in pediatric Non-Rhabdomyosarcoma soft tissue sarcomas: Consensus on surgical margin definition harmonization from the INternational Soft Tissue SaRcoma ConsorTium (INSTRuCT)

Background: Margin status following surgery in children, adolescents, and young adults with soft tissue sarcomas is controversial and has been defined differently by various specialties, with definitions changing over time and by cooperative group. The International Soft Tissue Sarcoma Consortium (INSTRuCT) is a collaboration of the Children's Oncology Group (COG) Soft Tissue Sarcoma Committee, European pediatric Soft Tissue sarcoma Study Group (EpSSG), and the European Cooperative Weichteilsarkom Studiengruppe (CWS) devoted to improving patient outcomes by pooling and mining cooperative group clinical trial data. Methods: The INSTRuCT non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) working group aimed to develop international harmonized recommendations regarding surgical margin assessment and definitions in children and adolescents with soft tissue tumors. Results and Conclusion: This review addresses accepted principles and areas of controversy, including the perspectives of surgeons, pathologists, radiation oncologists, and pediatric oncologists, to develop a framework for building common guidelines for future research

Rhabdomyosarcoma in Adults: New Perspectives on Therapy

Rhabdomyosarcoma (RMS) is well known as a pediatric disease. Most of the knowledge, like biology, genetics, and treatments of this disease, comes from studies done in that age group. The two subtypes of RMS, embryonic RMS and alveolar RMS, that affect mainly the pediatric population are well described in the literature and that has had an impact on the improvement in overall survival during the past 20 years. RMS in the adult population has a low incidence, therefor the study of RMS in this group is challenging. Pleomorphic RMS is the subtype that mainly affects adults and its biology and genetics are not yet completely understood and described. The risk factors for this tumor and the differences among adults and children is also poorly understood. The treatments for adults that have RMS are not standardized having an impact on the overall survival. Pleomorphic RMS has, compared to other adult sarcomas, poor overall survival. Adult patients with RMS have poor prognosis. The standardization of treatments for the adult population is necessary as maybe new treatments for this specific group. There are new treatment options that are being studied mostly in pediatrics and young adults. Immunotherapy is currently proposed as an important treatment possibility including different techniques like vaccination, antigen-mediated therapy, and immune checkpoints. Even if we have a better understanding of RMS, there are still unanswered questions. The improvements seen in the pediatric population are encouraging, but there is still the need to enhance better therapies for adults with RMS