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儿童肿瘤学组 2023 年研究蓝图:骨肿瘤。

Children's Oncology Group's 2023 blueprint for research: Bone tumors.

机构信息

Department of Individualized Cancer Management, Moffitt Cancer Center, Tampa, Florida, USA.

Division of Oncology, Children's Hospital of Philadelphia Research Institute, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

出版信息

Pediatr Blood Cancer. 2023 Sep;70 Suppl 6(Suppl 6):e30583. doi: 10.1002/pbc.30583. Epub 2023 Jul 27.

Abstract

The Children's Oncology Group (COG) Bone Tumor Committee is responsible for clinical trials and biological research on localized, metastatic, and recurrent osteosarcoma and Ewing sarcoma (EWS). Results of clinical trials in localized disease completed and published in the past 10 years have led to international standard-of-care chemotherapy for osteosarcoma and EWS. A recent focus on identifying disease subgroups has led to the identification of biological features associated with poor outcomes including the presence of circulating tumor DNA (ctDNA) at diagnosis, and specific genomic alterations-MYC amplification for osteosarcoma and STAG2 and TP53 mutation for EWS. Studies validating these potential biomarkers are under way. Clinical trials evaluating the addition of multitargeted kinase inhibitors, which are active in relapsed bone sarcomas, to standard chemotherapy are under way in osteosarcoma and planned in EWS. In addition, the Committee has data analyses and a clinical trial under way to evaluate approaches to local management of the primary tumor and metastatic sites. Given the rarity of bone sarcomas, we have prioritized international interactions and are in the process of forming an international data-sharing consortium to facilitate refinement of risk stratification and study of rare disease subtypes.

摘要

儿童肿瘤学组(COG)骨肿瘤委员会负责局部、转移性和复发性骨肉瘤和尤文肉瘤(EWS)的临床试验和生物学研究。过去 10 年完成并发表的局部疾病临床试验结果导致了骨肉瘤和 EWS 的国际标准化疗。最近的重点是确定疾病亚组,导致了与不良预后相关的生物学特征的鉴定,包括在诊断时存在循环肿瘤 DNA(ctDNA),以及特定的基因组改变——骨肉瘤中的 MYC 扩增和 EWS 中的 STAG2 和 TP53 突变。正在进行验证这些潜在生物标志物的研究。正在进行评估在标准化疗中添加多靶点激酶抑制剂(在复发性骨肉瘤中有效)的临床试验,用于骨肉瘤,并计划用于 EWS。此外,委员会正在进行数据分析和临床试验,以评估原发性肿瘤和转移部位的局部管理方法。鉴于骨肉瘤的罕见性,我们优先考虑国际合作,并正在组建一个国际数据共享联盟,以促进风险分层的细化和罕见疾病亚型的研究。

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Pediatr Blood Cancer. 2023 Sep;70 Suppl 6(Suppl 6):e30583. doi: 10.1002/pbc.30583. Epub 2023 Jul 27.
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