背景:髓母细胞瘤(MB)是一种罕见的脑肿瘤,在研究主要集中的儿童中更常见。成人的治疗建议主要基于回顾性数据和儿科经验;然而,2个年龄组的分子特征和治疗耐受性不同.在成年人中,预后工具并不理想,晚期复发是典型的,长期后遗症仍未得到充分研究。治疗不适应分子分类的进展;因此,成年MB的存活率没有提高。
方法:2017年,美国国家癌症研究所(NCI)获得了癌症月球组织的支持,以通过NCI-CONNECT解决患有罕见中枢神经系统肿瘤的成年人的挑战和未满足的需求。一个在患者之间建立伙伴关系的计划,卫生保健专业人员,研究人员,和宣传组织。2019年11月25日,NCI-CONNECT召集了领先的临床医生和科学家参加研讨会,回顾研究进展。分享科学见解,并讨论成人MB的临床挑战。
结果:工作组确定了临床试验设计中未满足的需求,组织采集和测试,肿瘤建模,和临床结果的测量。
结论:参与者确定了合作的机会;讨论了创建临床医生工作组的计划,研究人员,和患者倡导者;并制定了具体的行动项目,以加快成人MB的进展。
BACKGROUND: Medulloblastoma (MB) is a rare brain tumor occurring more frequently in children in whom research has been primarily focused. Treatment recommendations in adults are mainly based on retrospective data and pediatric experience; however, molecular features and treatment tolerance differ between the 2 age groups. In adults, prognostic tools are suboptimal, late recurrences are typical, and long-term sequelae remain understudied. Treatment has not adapted to molecular classification advances; thus, the survival rate of adult MB has not improved.
METHODS: In 2017, the National Cancer Institute (NCI) received support from the Cancer Moonshot℠ to address the challenges and unmet needs of adults with rare central nervous system tumors through NCI-CONNECT, a program that creates partnerships among patients, health care professionals, researchers, and advocacy organizations. On November 25, 2019, NCI-CONNECT convened leading clinicians and scientists in a workshop to review advances in research, share scientific insights, and discuss clinical challenges in adult MB.
RESULTS: Working groups identified unmet needs in clinical trial design, tissue acquisition and testing, tumor modeling, and measurement of clinical outcomes.
CONCLUSIONS: Participants identified opportunities for collaboration; discussed plans to create a working group of clinicians, researchers, and patient advocates; and developed specific action items to expedite progress in adult MB.