Recently published in Blood, the DREPAGREFFE-2 trial provides crucial insights into long-term cognitive and cerebrovascular outcomes in children with sickle cell anemia (SCA) and a history of abnormal cerebral velocities. This study follows up on the initial DREPAGREFFE-1 findings, which compared allogeneic stem cell transplantation (alloSCT) vs. standard-of-care (SoC) for stroke prevention in SCA.
Key Findings at 10 Years:
AlloSCT significantly reduced the progression of silent cerebral infarcts (SCI), whereas new SCI developed in patients under SoC.
Cognitive benefits were observed with higher working memory and processing speed index scores in the alloSCT group.
Quality of life improvements were notable in physical, school, and social functioning for patients who underwent transplantation.
These results reinforce the long-term neurological and quality-of-life benefits of alloSCT in pediatric SCA patients. With silent strokes being a major concern in this population, these findings could shape future treatment strategies and decision-making for high-risk children.
A huge thank you to Françoise Bernaudin and the research team for their dedication to advancing treatment options for children with SCA.
For more details, check the full study: https://lnkd.in/eCxxBzg8
