Childhood Cancer Survivorship Study

摘要 儿童癌症生存研究(CCSS)是一项多机构、多学科的合作研究 为系统评估被诊断为癌症的儿童的长期结果而建立的资源 在确诊后存活了五年或更长时间。随着成功的招聘和纵向跟进， 包括三十年(1970-1999)诊断和治疗的幸存者，CCSS是世界上 最大的已建立的生存研究开放资源，有38,036名合格的幸存者可供 晚期死亡率调查，以及25,665名对健康相关和生活质量有贡献的参与者 结果。该资源包括治疗暴露的全面注释、持续的纵向 随访和建立的生物库，其中8,380人的基因分型(SNP阵列)和DNA测序 幸存者可以被调查人员用来确定疾病的遗传易感性-以及治疗- 相关的后遗症。研究界的广泛使用已导致：出版或出版了381篇 手稿被引用超过26,500次；347篇提交摘要；59项由研究人员发起的赠款资助， 总计6700万美元的资金；由1225名调查人员组成的不同小组利用，其中包括91名早期职业生涯 受训者；进行11项随机试验；增加知识，为基于暴露的临床护理提供信息 准则；以及多个国家和国际合作倡议的非常成功的模式 儿科癌症存活期研究。在未来五年内，各项活动将确保中央支助系统的运作 通过维护、加强和促进其使用，将其作为一种强大和多产的资源。我们将扩大 收集数据以评估随年龄增长的生理和神经认知功能，表征加速衰老， 并在幸存者进入第四、第五和第六阶段时调查他们衰老的潜在病理生理学 几十年的生命。为针对与年龄相关的结果的家庭干预试验开发人口资源 将对1000名幸存者进行功能性能评估和标本采集。 此外，我们会透过以下途径，促进医疗服务研究的进行，从而加强医疗服务支援计划的资源 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。为了最大限度地扩大研究社区对CCSS资源的访问和使用，我们将 利用基于云的数据共享平台，开发具有数据访问工具的数据分析生态系统， 基因、治疗和表型/结果数据的可视化和分析。在未来，后期的评估 新疗法(免疫疗法、靶向/生物疗法、质子辐射疗法)的结果将是至关重要的。 因此，在NCI的监督下，CCSS将为未来的扩张制定全面的计划。这些倡议 将加强和推动CCSS进一步实现CCSS资源的总体目标，以增加 开展与儿科癌症存活期相关的创新性和高影响力研究。

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.