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/. 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.

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CSSCD was a multicenter, prospective study on the natural history of sickle cell disease and participant enrollment into Phase 1 of the CSSCD began in 1978. Participant entry ended in 1981 for all patients greater than six months of age; however, infants continued to be enrolled until 1988. Both mild and hospital-based sickle cell patients were recruited. A total of 4,085 participants, ranging in age from newborns to adults, were enrolled in Phase 1 from 23 centers across the US. Data collection for phase 1 of the CSSCD ended in 1988.For Phases 2 and 3, 450 were enrolled in the Phase 2 followup along with 17 children that were between 6 and 10 months of age at the time of Phase 1 enrollment. Phase 2 followup concluded in 1994 and 378 patients continued to be followed in the Phase 3 continuation. Data collection ended in 1998.
A total of 359 adult phase 1 participants were enrolled in phase 2A of the study between September of 1989 and July 1991. Exit visits began approximately 2 years later and were concluded in September of 1993.

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镰状细胞病合作研究（Cooperative Study of Sickle Cell Disease，以下简称CSSCD）于1977年启动，旨在阐明镰状细胞病（sickle cell disease, SCD）从出生到死亡的自然病程，以明确与该病发病率和死亡率相关的影响因素。其具体研究目标包括：1）探究镰状细胞病对婴幼儿至青春期生长发育的影响；2）分析可能与疼痛危象发作相关的诱因或事件；3）收集镰状细胞病主要并发症的性质、持续时长及转归相关数据；4）明确镰状细胞病所致器官损伤的特征、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工作。镰状细胞病合作研究第1阶段共纳入709名月龄不足6个月的婴儿，CSSCD第2、3阶段计划对该队列儿童进行为期10年的追加随访。该阶段的研究目标包括：1）前瞻性地明确镰状细胞病的自然病程；2）明确认知与学业功能和磁共振成像（magnetic resonance imaging, MRI）评估的脑部状态之间的关联；3）确定无症状性脑梗死的认知或行为标志物；4）分析家庭环境量表（Family Environment Scale, FES）所评估的家庭功能与脑部状态、认知功能及社会人口学因素之间的关联；5）延续相关研究，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阶段旨在探究1979年3月至1981年5月期间纳入第1阶段研究的成人队列患者（1956年1月1日前出生）的心、肺、肾及肝脏器官损伤的进展情况。来自11个研究中心的共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BioData Catalyst平台（https://biodatacatalyst.nhlbi.nih.gov/resources/data/）查询。申请人需在dbGaP中提交数据访问申请，获批后即可在BioData Catalyst平台获取所申请的数据。若需了解数据可用性相关问题，可通过https://biodatacatalyst.nhlbi.nih.gov/contact联系BioData Catalyst团队。
CSSCD是一项针对镰状细胞病自然病程的多中心前瞻性研究，其第1阶段的受试者招募工作于1978年启动。1981年，所有月龄大于6个月的受试者招募工作宣告结束，但婴儿受试者的招募工作持续至1988年。研究同时招募轻症镰状细胞病患者及住院患者。第1阶段共从全美23个研究中心纳入4085名受试者，年龄跨度从新生儿至成人。CSSCD第1阶段的数据采集工作于1988年结束。
在第2、3阶段中，共纳入450名受试者进行第2阶段随访，其中包括17名在第1阶段入组时年龄为6~10个月的儿童。第2阶段随访工作于1994年结束，其中378名患者继续接受第3阶段随访。第3阶段的数据采集工作于1998年结束。
1989年9月至1991年7月期间，共有359名第1阶段的成人受试者纳入本研究的2A阶段。随访收尾访视工作于约2年后启动，并于1993年9月全部完成。