Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.
Age range
4 Years – 65 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Measurement of longitudinal change in FEV1
Timeframe: Through study completion, an average of four years
Percent predicted value of longitudinal change in FEV1
Timeframe: Through study completion, an average of four years
Measurement of longitudinal change in FVC
Timeframe: Through study completion, an average of four years
Percent predicted value of longitudinal change in FVC
Timeframe: Through study completion, an average of four years
FEV1/FVC Ratio Percentage
Timeframe: Through study completion, an average of four years
Longitudinal change in eGFR
Timeframe: Through study completion, an average of four years
Longitudinal change in albuminuria levels
Timeframe: Through study completion, an average of four years
Longitudinal change in TRJV in adults with SCD treated with nonmyeloablative allo HSCT in adults
Timeframe: Through study completion, an average of four years
Longitudinal change in SBP/DBP in adults with SCD treated with nonmyeloablative allo HSCT in adults
Timeframe: Through study completion, an average of four years