Cooperative Study of Sickle Cell Disease (CSSCD)

The Cooperative Study of Sickle Cell Disease was initiated in 1977 to determine the natural history of sickle cell disease (SCD) from birth to death in order to identify those factors contributing to the morbidity and mortality of the disease. Specific objectives included: 1) to study the effect of sickle cell disease on growth and development from birth through adolescence 2) to study the conditions or events that may be related to the onset of painful crises 3) to obtain data on the nature, duration, and outcome of major complications of SCD 4) determine the nature, prevalence, and age- related incidence of organ damage due to SCD, and 5) study the role of SCD and its interaction with selected health events.

Phases 2 and 3 of the study involved followup of the infant cohort. A total of 709 infants (age less than 6 months) were enrolled during Phase 1 of the Cooperative Study of Sickle Cell Disease (CSSCD), and Phases 2 and 3 of the CSSCD was designed to follow these children for an additional 10 years. The study objectives included: 1) define prospectively the natural history of sickle cell disease; 2) determine the relationships between cognitive and academic functioning and brain status as determined by MRI; 3) determine the cognitive or behavioral markers of silent infarct; 4) determine the relationship of family functioning on the Family Environment Scale (FES) to brain status, cognitive functioning, and social and demographic factors; 5) continue studies that will enhance the state of knowledge on the influence of sickle cell disease on the psychosocial adjustment of children and adolescents.

Phase 2A of the study sought to examine the progression of organ damage in the heart, lung, kidney, and liver in adult cohort patients (born before 1/1/56) enrolled in phase 1 of the study between 3/79 and 5/81. A total of 620 patients from 11 centers were eligible for phase 2A.

Instructions for requesting individual-level data are available on BioData Catalyst at [https://biodatacatalyst.nhlbi.nih.gov/resources/data/](https://biodatacatalyst.nhlbi.nih.gov/resources/data/). Apply for data access in dbGaP. Upon approval, users may begin accessing requested data in BioData Catalyst. For questions about availability, you may contact the BioData Catalyst team at [https://biodatacatalyst.nhlbi.nih.gov/contact](https://biodatacatalyst.nhlbi.nih.gov/contact).

镰状细胞病合作研究（Cooperative Study of Sickle Cell Disease, CSSCD）于1977年启动，旨在明确镰状细胞病（sickle cell disease, SCD）从出生到死亡的自然病程，以识别影响该病发病率与死亡率的相关因素。具体研究目标包括：1）探究镰状细胞病从出生至青春期对生长发育的影响；2）研究可能与疼痛危象发作相关的状况或事件；3）获取镰状细胞病主要并发症的性质、持续时间与转归相关数据；4）明确镰状细胞病所致器官损伤的性质、患病率及年龄相关发病率；5）研究镰状细胞病的作用及其与特定健康事件的相互作用。

本研究的第2、3阶段针对婴儿队列开展随访。镰状细胞病合作研究（CSSCD）第1阶段共纳入709名月龄不足6个月的婴儿；CSSCD第2、3阶段计划对这些儿童进行为期10年的追加随访。本阶段研究目标包括：1）前瞻性明确镰状细胞病的自然病程；2）明确认知与学业功能及磁共振成像（magnetic resonance imaging, MRI）所评估的脑状态之间的关联；3）明确无症状性脑梗死的认知或行为标志物；4）明确家庭环境量表（Family Environment Scale, FES）所反映的家庭功能与脑状态、认知功能及社会人口学因素之间的关联；5）持续开展相关研究，以深化对镰状细胞病对儿童及青少年社会心理适应影响的认知。

本研究的2A阶段旨在考察1979年3月至1981年5月期间纳入镰状细胞病合作研究第1阶段的成人队列患者（出生于1956年1月1日之前）的心、肺、肾及肝脏器官损伤的进展情况。共有来自11个研究中心的620名患者符合2A阶段的入组标准。

个体水平数据的申请指南可在生物数据催化平台（BioData Catalyst）查阅：https://biodatacatalyst.nhlbi.nih.gov/resources/data/。申请者需在dbGaP申请数据访问权限。获批后，用户即可在生物数据催化平台获取所申请的数据。若对数据可用性有疑问，可通过https://biodatacatalyst.nhlbi.nih.gov/contact联系生物数据催化平台团队。