1. In this nonrandomized controlled intervention study, matched sibling donor stem cell transplantation (MSD-HSCT) was associated with lower transcranial doppler velocities (TCDs) at 1 year compared to chronic transfusions.
2. MSD-HSCT was also associated with changes in highest TCDs at 3 years, normalization rate at 1 year, and ferritin levels at 1 and 3 years.
Evidence Rating Level: 1 (Excellent)
Study Rundown: Increased transcranial doppler velocity (TCD) in children with sickle cell anemia (SCA) is associated with an increased risk of ischemic stroke, stenosis, and silent cerebral infarcts. Previous studies have identified the pivotal role of chronic transfusions in reducing stroke risk, while newer studies have considered other preventive approaches such as hydroxyurea or matched sibling donor stem cell transplantation (MSD-HSCT). In this randomized control trial, children with SCA who received MSD-HSCT had lower TCDs at 1 and 3 years compared to those receiving care as usual (chronic transfusions). MSD-HSCT also led to greater normalization rates at 1 year and lower ferritin levels at 1 year and 3 years.
Overall, this study shows that children with SCA that receive MSD-HSCT have significantly lower TCDs at 1 year. The study is limited by its use of a short-term endpoints (cerebral velocities at 1 year), non-randomized design, and relatively small sample size. Further research is required to assess the effects of MSD-HSCT on clinical outcomes.
Click to read the study in JAMA
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