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Overview
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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.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.
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Outcomes
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1. To follow prospectively newborns with sickle cell disease for a minimum of 10 years, and elucidate issues relating to development, infections, efficacy of prophylactic regimens, and onset and course of acute and chronic complications, including abnormal pulmonary function and gallstones
2. To accurately determine incidence rates and obtain data on the nature, duration and outcome of selected acute special events.
3. Prevalence and incidence, age of onset, rate of progression, and outcome of chronic organ damage.
4. The role of sickle cell disease and its interaction with selected health events. Data will continue to be collected on pregnancy, surgery with general anesthesia, and infection.
5. Economic, educational and vocational levels, and obtain data on other psychosocial aspects including self-concept, coping, and intra-family relationships.
6. To develop a data base which will be used to evaluate treatment modalities, describe the spectrum of diseases and establish criteria for potential therapeutic interventions.
7. To further elucidate previously unrecognized clinical manifestations and their sequelae, abnormal pulmonary function and aseptic necrosis.
8. To conduct in-depth investigations relating to the wide variability of clinical manifestations in sickle cell disease and delineate factors contributing to the spectrum of mild to severe disease. Studies to be prioritized from gene mapping and on a subset of patients (i.e., patients over 40 years, sib-sib pairs, parent –child pairs), cellular heterogeneity, quantitation of Hb S polymerization, kinetics of Hb S gelation, and adherence of erythrocytes to vascular endothelium.
9. Prophylactic penicillin trial in the pediatric age group to evaluate its efficacy in preventing severe overwhelming infections.
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