Childhood Cancer Survivorship Study

摘要 儿童癌症幸存者研究（CCSS）是一项多机构、多学科的合作研究 建立了一个资源，以系统地评估被诊断患有癌症的儿童的长期结果， 从诊断开始存活了五年或更长时间。随着成功招募和纵向跟踪的 包括三十年（1970-1999）诊断和治疗的幸存者的队列，CCSS是世界上 最大的幸存者研究开放资源，有38，036名合格的幸存者可供使用， 对晚期死亡率的调查，以及25，665名参与者对健康相关和生活质量的贡献 结果。该资源包括对治疗暴露的全面注释， 随访和建立的生物储存库，从基因型（SNP阵列）和DNA测序的8，380 幸存者可供研究人员鉴定疾病的遗传易感性-和治疗- 相关的后期影响。被研究界广泛使用的结果是： 手稿现在被引用超过26，500次; 347份摘要; 59份赞助者发起的赠款， 总计6700万美元的资金;由1,225名调查人员组成的多元化小组使用，其中包括91名早期职业生涯 受训人员;进行11项随机试验;增加知识，以告知基于医疗保健的临床护理 指导方针;以及多个国家和国际合作倡议的一个非常成功的模式， 儿童癌症存活率研究在今后五年中，将开展活动，确保哥斯达黎加社会保障机构的运作 作为一种强大的生产性资源，保持，加强和促进其使用。我们将扩大 收集数据以评价生理和神经认知功能随老化的变化，表征加速老化， 并调查幸存者在进入第四、第五和第六个阶段时衰老的潜在病理生理学。 几十年的生活。为针对年龄相关结局的干预试验开发人口资源， 将在1000名幸存者的子队列中进行功能性能评估和样本收集。 此外，我们会透过下列方法，促进医疗服务研究，以增加“顾客支援计划”的资源： 收集数据以评估患者、提供者和医疗保健系统因素及其与访问的关联， 质量和护理成本。为使研究界能尽量取得和使用CCSS的资源，我们会 利用基于云的数据共享平台，并开发一个具有数据访问工具的数据分析生态系统， 遗传、治疗和表型/结果数据的可视化和分析。在未来，评估后期 新疗法（免疫疗法、靶向/生物疗法、质子束放射疗法）的结果至关重要。 因此，在NCI的监督下，哥斯达黎加社会保障局将为未来的扩展制定一个全面的计划。这些举措 将加强和促进CCSS，以进一步实现CCSS资源的总体目标， 开展与儿童癌症生存率相关的创新和高影响力研究。

ABSTRACT The Childhood Cancer Survivor Study (CCSS) is a multi-institutional, multi-disciplinary collaborative research resource established to systematically evaluate long-term outcomes among children diagnosed with cancer who survived five or more years from diagnosis. With the successful recruitment and longitudinal follow-up of the cohort that includes survivors diagnosed and treated over three decades (1970-1999), the CCSS is the world's largest established open resource for survivorship research with 38,036 eligible survivors available for investigation of late mortality, and 25,665 participants who have contributed health-related and quality of life outcomes. The resource includes comprehensive annotation of treatment exposures, ongoing longitudinal follow-up and an established biorepository from which genotype (SNP array) and DNA sequencing of 8,380 survivors are available to investigators for identification of genetic susceptibility for disease- and treatment- related late effects. Extensive use by the research community has resulted in: 381 published or in press manuscripts now cited over 26,500 times; 347 presented abstracts; 59 investigator-initiated grants funded, totaling $67 million in funding; utilization by a diverse group of 1,225 investigators including 91 early career trainees; conduct of 11 randomized trials; increased knowledge to inform exposure-based clinical care guidelines; and a highly successful model for multiple national and international collaborative initiatives of pediatric cancer survivorship research. During the next five years, activities will ensure the functioning of CCSS as a strong and productive resource by maintaining, enhancing and promoting its use. We will expand the collection of data to evaluate physiologic and neurocognitive function with aging, characterize accelerated aging, and investigate underlying pathophysiology of aging of survivors as they enter into their fourth, fifth and sixth decades of life. To develop a population resource for intervention trials targeting age-related outcomes, in-home functional performance assessment and specimen collection will be performed in a sub-cohort of 1000 survivors. Further, we will enhance the CCSS resource by facilitating the conduct of health services research through collection of data to evaluate patient, provider, and health care system factors and their associations with access, quality, and cost of care. To maximize the research community's access and use of the CCSS resource, we will leverage a cloud-based data sharing platform and develop a data analysis ecosystem with tools for data access, visualization and analysis of genetic, treatment and phenotypic/outcome data. In the future, assessment of late outcomes of novel therapies (immunotherapy, targeted/biologic, proton beam radiation) will be essential. Therefore, with NCI oversight, CCSS will formulate a comprehensive plan for future expansion. These initiatives will enhance and promote CCSS to further achieve the overarching goal of the CCSS resource to increase the conduct of innovative and high impact research related to pediatric cancer survivorship.