Chen Celine, Dorado Garcia Heathcliff, Scheer Monika, Henssen Anton G
Department of Pediatric Oncology/Hematology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Pediatrics 5, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany.
Front Oncol. 2019 Dec 20;9:1458. doi: 10.3389/fonc.2019.01458. eCollection 2019.
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children, and can be subcategorized histologically and/or based on PAX-FOXO1 fusion gene status. Over the last four decades, there have been no significant improvements in clinical outcomes for advanced and metastatic RMS patients, underscoring a need for new treatment options for these groups. Despite significant advancements in our understanding of the genomic landscape and underlying biological mechanisms governing RMS that have informed the identification of novel therapeutic targets, development of these therapies in clinical trials has lagged far behind. In this review, we summarize the current frontline multi-modality therapy for RMS according to pediatric protocols, highlight emerging targeted therapies and immunotherapies identified by preclinical studies, and discuss early clinical trial data and the implications they hold for future clinical development.
横纹肌肉瘤（RMS）是儿童中最常见的软组织肉瘤，可根据组织学分类和/或基于PAX-FOXO1融合基因状态进行亚分类。在过去的四十年中，晚期和转移性RMS患者的临床结局没有显著改善，这凸显了为这些患者群体寻求新治疗方案的必要性。尽管我们对RMS的基因组格局和潜在生物学机制的理解取得了重大进展，这些进展为新型治疗靶点的识别提供了依据，但这些疗法在临床试验中的开发却远远滞后。在本综述中，我们根据儿科方案总结了目前RMS的一线多模态治疗方法，强调临床前研究确定的新兴靶向治疗和免疫治疗，并讨论早期临床试验数据及其对未来临床开发的意义。
