The Cooperative Study of Sickle Cell Disease (CSSCD) Phase 1

Objectives:

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 include: 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.

Background:

Sickle cell disease is a single-gene defect that results in sickle-shaped red blood cells. Although the manifestations of sickle cell disease have been described, variations in the severity and number of manifestations, as well as interactions with other health events, leads to significant gaps in the understanding of the natural history of the disorder. For example, impairments to renal, cardiac, and pulmonary organ function are known to occur in sickle cell patients; however, the descriptions of these outcomes was generally retrospective in nature and occurred when organ damage was severe. In addition, only limited data existed on the social, economic, educational, vocational, and psychological adjustment of patients and families, and as with any chronic disease, impediments to sickle cell patients achieving their educational and vocational goals needs to be elucidated.

Subjects:

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.

Design:

Four protocols were developed according to participant age: Newborn to six months, pediatric (6 months to 10 years of age), adolescent (age 10-19 years) and adult (age 20+). Protocols were designed to collect similar data at similar times. Participants underwent a baseline exam for assessment of demographics, prior medical history, lab assessments, and clinical data. Post baseline data included routine follow-up examinations, measures of organ damage, and collection of acute and chronic complications.