Dear Colleagues,

Rhabdomyosarcoma (RMS) is a rare mesodermal tumor that can occur at any age but which is mostly diagnosed in children and adolescents. The cure rates for RMS have increased from 25% in the 1970s to 70% in 1990 due to the efforts of cooperative groups on both sides of the Atlantic. This improvement has mainly been achieved in children and adolescents who were eligible for pediatric clinical trials. Young and elderly adults still have poor outcomes. Similarly, no significant progress has been achieved in patients with metastatic disease. Four major RMS histologic subtypes exist: embryonal, alveolar, pleomorphic, and spindle cell RMS. In the majority of alveolar RMS fusion genes, PAX3/7-FOXO1 are detected. However, there is little known regarding phenotypic and biological heterogeneity across the RMS spectrum, especially for fusion-negative RMS. The recent risk grouping system used to adapt the therapy burden is based on clinical features, pathological subtypes, and PAX3/7-FOXO1 fusion status. However, the stratification parameters do not include the full range of the genetic variability of RMS.

The long-term sequelae remain a major problem in survivors. The current trend is to deescalate chemotherapy and radiotherapy treatment, as many trials in the past have failed to show improved outcomes by increasing the dose administered or providing additional drugs. Special consideration must be given to developing better cooperation with medical oncologists, including young adults in pediatric trials, and developing treatment recommendations for adult patients with biologically different RMS, including recommendations for the lifelong surveillance of survivors of RMS.

For this Special Issue, we welcome original and review papers focusing on the understanding of the genomic landscape and underlying biological mechanisms to identify objective biomarkers for the better stratification and monitoring of therapy and the identification of novel therapeutic targets and therapies. We look also for papers on the late effects and quality of life of RMS long-term survivors in the context of reducing the sequelae through the use of optimal-risk adapted local and systemic therapy. Articles providing solutions for developing overarching trials for all ages are also welcome.

We look forward to receiving your contributions.

Prof. Dr. Ewa Koscielniak
Dr. Monika Sparber-Sauer
Guest Editors

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• rhabdomyosarcoma
• molecular characterization
• late effects
• RMS predisposition
• innovative therapies