U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
Vanderbilt University Medical Center
Summary
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.
Description
Our primary objective is initiating a personalized approach to curative therapies in children and adults with sickle cell disease (SCD) to maximize benefits and limit adverse outcomes. Limited clinical studies exist to determine the long-term health outcomes following curative therapies for SCD. With emerging curative therapies for SCD (allogeneic \[allo\] hematopoietic stem cell transplant \[HSCT\], gene therapy/editing), long-term health outcomes studies are critical to inform personalized choices. Unfortunately, adverse outcomes have started to emerge after SCD curative therapy. Thus, risks…
Eligibility
- Age range
- 4–65 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria * Confirmed laboratory diagnosis of SCD * Ability to give informed consent * Ability to provide pre- and post-curative therapy data * Treated with either one HSCT or with standard disease-modifying therapy Exclusion Criteria •History of non-compliance
Locations (5)
- Children's National Medical CenterWashington D.C., District of Columbia
- Emory University School of MedicineAtlanta, Georgia
- Johns Hopkins HospitalBaltimore, Maryland
- National Institutes of Health Clinical CenterBethesda, Maryland
- Vanderbilt University Medical CenterNashville, Tennessee