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儿童肿瘤学组 2023 年研究蓝图:神经母细胞瘤。

Children's Oncology Group's 2023 blueprint for research: Neuroblastoma.

机构信息

Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.

出版信息

Pediatr Blood Cancer. 2023 Sep;70 Suppl 6(Suppl 6):e30572. doi: 10.1002/pbc.30572. Epub 2023 Jul 17.

Abstract

Neuroblastoma is the most common extra-cranial solid tumor in children and is known for its clinical heterogeneity. A greater understanding of the biology of this disease has led to both improved risk stratification and new approaches to therapy. Outcomes for children with low and intermediate risk disease are excellent overall, and efforts to decrease therapy for such patients have been largely successful. Although survival has improved over time for patients with high-risk disease and treatments evaluated in the relapse setting are now being moved into earlier phases of treatment, much work remains to improve survival and decrease therapy-related toxicities. Studies of highly annotated biobanked samples continue to lead to important insights regarding neuroblastoma biology. Such studies, along with correlative biology studies incorporated into therapeutic trials, are expected to continue to provide insights that lead to new and more effective therapies. A focus on translational science is accompanied by an emphasis on new agent development, optimized risk stratification, and international collaboration to address questions relevant to molecularly defined subsets of patients. In addition, the COG Neuroblastoma Committee is committed to addressing the patient/family experience, mitigating late effects of therapy, and studying social determinants of health in patients with neuroblastoma.

摘要

神经母细胞瘤是儿童中最常见的颅外实体瘤,其临床表现具有异质性。对该疾病生物学特性的深入了解,不仅改善了风险分层,还为治疗提供了新的方法。低危和中危患儿的总体预后良好,减少此类患者治疗的努力已基本取得成功。虽然高危患儿的生存率随着时间的推移有所提高,且在复发环境中评估的治疗方法现已被用于更早的治疗阶段,但仍有许多工作要做,以提高生存率并减少与治疗相关的毒性。对高度注释的生物样本库样本的研究继续为神经母细胞瘤生物学提供重要见解。这些研究,以及纳入治疗试验的相关生物学研究,有望继续提供新的、更有效的治疗方法的见解。对转化科学的关注伴随着对新药物开发、风险分层优化以及国际合作的重视,以解决与患者分子定义亚组相关的问题。此外,COG 神经母细胞瘤委员会致力于解决患者/家庭的体验、减轻治疗的后期影响以及研究神经母细胞瘤患者健康的社会决定因素。

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