Better 10-Year Cerebrovascular Outcome After Transplant Than on Standard-Care in Sickle Cell Anemia: DREPAGREFFE Trial - PubMed
4 hours ago
- #sickle cell anemia
- #cerebrovascular outcomes
- #hematopoietic cell transplantation
- The DREPAGREFFE trial compared hematopoietic cell transplantation (HCT) from a matched sibling donor (MSD) versus standard-care (chronic transfusion or hydroxyurea) in sickle cell anemia children with cerebral vasculopathy.
- At 10-year follow-up, MSD-HCT showed superior outcomes: no deaths or strokes occurred in either group, but HCT resulted in significantly fewer hospitalizations, crises, and better quality of life.
- In stroke-free patients, stenosis and silent cerebral infarct scores decreased significantly more after HCT, and cognitive functions like working memory and processing speed improved, whereas they worsened under standard-care.
- The study concludes that HCT should be considered earlier for eligible children to preserve neurological function and improve long-term quality of life.