About this study
Sickle cell disease (SCD) is a group of blood disorders that affects how red blood cells carry oxygen. Red blood cells use hemoglobin to carry oxygen to tissues in the body. Normal red blood cells are round and flexible and can flow through the blood vessels. In people with SCD, the red blood cells are stiff and shaped like bananas (sickle-shaped). These cells stick together. They can clog the blood vessels, causing pain and in some cases, organ damage.
The standard care for SCD includes medicines and blood transfusions to control pain and other health problems. For more severe cases, a patient might get a stem cell (bone marrow) transplant. This treatment replaces a patient’s unhealthy blood cells with healthy ones from a donor.
This study will compare patients who get standard care with those who get stem cell transplants for SCD. The researchers want to know how standard treatments and stem cell transplants affect the quality of life for children, teens, and young adults with SCD.
Researchers will collect information about patients’ well-being, pain levels, and thinking abilities. This information will be collected from:
- Patients’ medical records
- Yearly surveys about their health and quality of life
- Yearly cognitive (thinking) tests to measure how well their brain works
The study will last 3 years.
The goal of this study is to understand how well people with sickle cell disease do after a stem cell transplant, as compared to those who get the standard treatment with medicines and blood transfusions.
Eligibility overview
- Sickle cell disease
- Not getting a stem cell transplant
- Ages 3–20
- Enrolled in the GRNDaD study