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Better 10-Year Cerebrovascular Outcome After Transplant Than on Standard-Care in Sickle Cell Anemia: DREPAGREFFE

Francoise Bernaudin1,2, Suzanne Verlhac1,3,4, Elisabeth Ducros-Miralles5

  • 1Referral Center for Sickle Cell Disease, CHIC Hospital, Créteil, France.

American Journal of Hematology
|May 29, 2026
PubMed

Insights

Hematopoietic cell transplantation (HCT) in children with sickle cell anemia (SCA) significantly improves quality of life and neurologic function compared to standard care. Long-term results show HCT reduces hospitalizations and crises, preserving cognitive abilities.

Area of Science:

  • Hematology
  • Neurology
  • Pediatrics

Background:

  • Sickle cell anemia (SCA) is associated with cerebral vasculopathy, increasing stroke risk.
  • Current management includes chronic transfusion (CT) or hydroxyurea, with hematopoietic cell transplantation (HCT) as an alternative.
  • Previous studies suggested benefits of match sibling donor (MSD)-HCT over standard care in SCA children, but long-term outcomes required further evaluation.

Purpose of the Study:

  • To evaluate the 10-year outcomes of match sibling donor (MSD)-HCT compared to standard care in children with sickle cell anemia (SCA) and cerebral vasculopathy.
  • To assess the impact of MSD-HCT on neurologic function, quality of life, and disease burden over a decade.

Main Methods:

  • A prospective, multicenter trial (DREPAGREFFE-2) followed 67 SCA children (5-15 years) initially enrolled in DREPAGREFFE-1.
  • Participants were randomized to either standard care (CT or hydroxyurea) or MSD-HCT.
  • Outcomes including neurologic status (vasculopathy, silent cerebral infarcts), cognitive performance, quality of life, and disease-related events were assessed at 10 years.

Main Results:

  • No deaths or strokes occurred in either group over 10 years.
  • MSD-HCT significantly improved quality of life, reduced hospitalizations, hospitalized days, and crises compared to standard care.
  • MSD-HCT led to significant improvements in working memory and processing speed, and a decrease in stenosis and silent cerebral infarct scores, effects not seen with standard care.

Conclusions:

  • Hematopoietic cell transplantation (HCT) using a match sibling donor (MSD) offers significant long-term benefits for children with sickle cell anemia (SCA), including improved neurologic function and quality of life.
  • The findings support earlier consideration of MSD-HCT for SCA children to prevent long-term neurologic sequelae and enhance future productivity.
  • Long-term follow-up revealed cognitive and neuroimaging benefits of MSD-HCT that were not apparent in shorter-term analyses.

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