横纹肌肉瘤风险分层的最新进展及当前和未来儿童肿瘤学组临床试验的原理。
An update on rhabdomyosarcoma risk stratification and the rationale for current and future Children's Oncology Group clinical trials.
机构信息
Division of Oncology, Hyundai Cancer Institute, Children's Hospital Orange County, Orange, California, USA.
Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USA.
出版信息
Pediatr Blood Cancer. 2022 Apr;69(4):e29511. doi: 10.1002/pbc.29511. Epub 2022 Feb 7.
Abstract
Children and adolescents with rhabdomyosarcoma (RMS) comprise a heterogeneous population with variable overall survival rates ranging between approximately 6% and 100% depending on defined risk factors. Although the risk stratification of patients has been refined across five decades of collaborative group studies, molecular prognostic biomarkers beyond FOXO1 fusion status have yet to be incorporated prospectively in upfront risk-based therapy assignments. This review describes the evolution of risk-based therapy and the current risk stratification, defines a new risk stratification incorporating novel biomarkers, and provides the rationale for the current and upcoming Children's Oncology Group RMS studies.
摘要
横纹肌肉瘤（RMS）患儿和青少年是一个异质性人群，总生存率因定义的风险因素而异，约为 6%至 100%。尽管经过五十年的协作组研究，患者的风险分层已经得到了完善，但除 FOXO1 融合状态之外的分子预后生物标志物尚未前瞻性地纳入基于风险的初始治疗分配中。本综述描述了基于风险的治疗方法的演变和当前的风险分层，定义了一个纳入新生物标志物的新风险分层，并为当前和即将开展的儿童肿瘤学组 RMS 研究提供了依据。
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