Objectives

The main purposes of this study were the following: 1) Establish a database to house medical information about a large number of children and adults with sickle cell disease over a long period of time. 2) To identify potential participants for future sickle cell disease clinical trials. 3) To collect information about how sickle cell disease affects quality of life. 4) To evaluate the relationship between sickle cells patients' characteristics and medical events.

Background

Sickle cell disease (SCD) is an inherited blood disorder. Symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." Symptoms vary considerably in frequency and severity between patients and within the same patient over time. However, the low prevalence of SCD, about 100,000 individuals in the United States, has limited progress in clinical research. The Collaborative Data Project (C-Data) was established to create a comprehensive database of children and adults receiving medical care at participating Comprehensive Sickle Cell Centers (CSCC) who would potentially be eligible for inclusion in SCD clinical trials.

Participants

Eligible participants included children and adults with a diagnosis of SCD who had been seen within 24 months prior to study entry in the hospital or clinical setting, and who were expected to return episodically or regularly for care at one of the CSCCs. The available data includes 3638 participants.

Design

The C-Data Project created a database with information collected from participants seen for medical care at a participating CSCC. Participant enrollment occurred either at the time of a special C-Data recruitment event or at a routine clinic visit. Information, such as medical history, was obtained from medical records for a period of at least 5 years. Once a year, participants took part in a short interview about their health and lifestyle. Participants also completed quality-of-life and patient satisfaction questionnaires during the first year of the study, with some participants completing additional questionnaires. During an interview, participants were asked general questions about their experience with SCD and more specific questions on headaches and any possible relation they may have to SCD pain. CSCC researchers used the information in the database to perform analyses and to contact participants about enrolling in SCD clinical trials.

Conclusions

The study demonstrated that a large-scale, multi-center database could be established to track medical information from both children and adults with SCD. The project concluded that centralized data collection is essential for the rapid enrollment of participants in clinical trials. Analysis of the C-Data confirmed the high degree of phenotypic diversity in SCD, showing that patients' characteristics and medical events (such as pain crises and organ damage) vary significantly, even among those with the same genotype.

Ballas SK, Lieff S, Benjamin LJ, et al. Definitions of the phenotypic manifestations of sickle cell disease. Am J Hematol. 2010;85(1):6-13. doi:10.1002/ajh.21550

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