Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial
Abstract
Management of cerebral vasculopathy in sickle cell anemia (SCA) includes standard-care, that is, chronic transfusion (CT) or hydroxyurea, and hematopoietic cell transplantation (HCT). DREPAGREFFE-1 (December 2010/June 2013), a French multicenter trial, was the first prospective trial comparing standard-care to match sibling donor (MSD)-HCT in 67 (35F/32M) SCA children (5-15 year) on CT for abnormal time-averaged mean maximum velocities (TAMMV ≥ 200 cm/s). Seven had a stroke history. We reported that MSD-HCT reduced the highest TAMMVs at 1-and 3-year (p < 0.001) and improved quality of life (QoL) for physical and school functioning. In stroke-free patients, the 3-year stenosis score was lower (p = 0.010). Nevertheless, no significant difference was observed for silent cerebral infarcts (SCI) and cognitive performance. This prompted us to initiate DREPAGREFFE-2 to reevaluate the outcomes at 10 years (September 2022/August 2024) with the same 67 SCA children. No death or stroke occurred in either arm. No rejection or chronic-GvHD arose in the MSD-HCT group (n=32). In the standard-care group (n=35), 16 were on hydroxyurea, and 16 on CT at Year 10, and 3 received haploidentical-HCT. After MSD-HCT, the QoL was better, even for social functioning, and the number of hospitalizations, hospitalized days (p<0.001), and crises (p=0.001) was lower than on standardcare. In stroke-free patients, stenosis (p=0.027) and SCI scores (p=0.041) decreased significantly more after MSD-HCT than on standard-care; working memory (p=0.016) and processing speed (p=0.011) improved significantly after MSD-HCT, but worsened on standard-care. These effects were not previously detected with shorter follow-up. This supports earlier consideration of HCT for SCA children with MSD to preserve neurologic function and QoL for a more productive future.
Related
- Editorial: Impact of new therapeutic approaches for the treatment of post-transplant tumors on the outcome of the transplanted organ
- Better Outcome After Thyroplasty Type I in Unilateral Vocal Fold Paralysis Without Synkinesis and Synkinetically-Reinnervated Type IV Vocal Fold Paralysis
- Donor-Recipient Size Mismatch is a Critical Determinant of Kidney Transplant Outcome
- Heart Disease and Cerebrovascular Disease Mortality Trends, 2015–2025
- Editorial: Chronic large-vessel occlusion in cerebrovascular disease: pathophysiology, diagnosis, and therapeutic advances
BibTeX
@article{bernaudin2026better,
title = {{Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial}},
author = {Francoise Bernaudin and Suzanne Verlhac and Elisabeth Ducros‐miralles and Cécile Arnaud and Isabelle Genty and Marie Petras and Catherine Paillard and Gonzalo de Luna and Benoit Meunier and Isabelle Thuret and Annie Kamdem and Corinne Pondarré and Giovanna Cannas and Christelle Chantalat‐auger and Nadia Firah and Sophie Pertuisel and Laure Joseph and Florence Missud and Mariane de Montalembert and Corinne Guitton and Claire Pluchart and Valentine Brousse and Monique Elmaleh and Vincent Gajdos and Emmanuella Leveillé and Ekaterina Belozertseva and Mabel Gaba and Catherine Poirot and Virginie Barraud‐lange and Philippe Chadebech and France Pirenne and Myriam Bernaudin and Bénédicte Neven and Pierre Frange and Marie Angoso and Karima Yakouben and Bénédicte Bruno and Nathalie Dhédin and Jean‐hugues Dalle and Regis Peffault Delatour and Camille Jung},
journal = {American Journal of Hematology},
year = {2026},
month = may,
doi = {10.1002/ajh.70336},
url = {https://normandie-univ.hal.science/hal-05769867v1},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial
AU - Francoise Bernaudin
AU - Suzanne Verlhac
AU - Elisabeth Ducros‐miralles
AU - Cécile Arnaud
AU - Isabelle Genty
AU - Marie Petras
AU - Catherine Paillard
AU - Gonzalo de Luna
AU - Benoit Meunier
AU - Isabelle Thuret
AU - Annie Kamdem
AU - Corinne Pondarré
AU - Giovanna Cannas
AU - Christelle Chantalat‐auger
AU - Nadia Firah
AU - Sophie Pertuisel
AU - Laure Joseph
AU - Florence Missud
AU - Mariane de Montalembert
AU - Corinne Guitton
AU - Claire Pluchart
AU - Valentine Brousse
AU - Monique Elmaleh
AU - Vincent Gajdos
AU - Emmanuella Leveillé
AU - Ekaterina Belozertseva
AU - Mabel Gaba
AU - Catherine Poirot
AU - Virginie Barraud‐lange
AU - Philippe Chadebech
AU - France Pirenne
AU - Myriam Bernaudin
AU - Bénédicte Neven
AU - Pierre Frange
AU - Marie Angoso
AU - Karima Yakouben
AU - Bénédicte Bruno
AU - Nathalie Dhédin
AU - Jean‐hugues Dalle
AU - Regis Peffault Delatour
AU - Camille Jung
PY - 2026
DA - 2026/05/28
JO - American Journal of Hematology
DO - 10.1002/ajh.70336
UR - https://normandie-univ.hal.science/hal-05769867v1
AB - Management of cerebral vasculopathy in sickle cell anemia (SCA) includes standard-care, that is, chronic transfusion (CT) or hydroxyurea, and hematopoietic cell transplantation (HCT). DREPAGREFFE-1 (December 2010/June 2013), a French multicenter trial, was the first prospective trial comparing standard-care to match sibling donor (MSD)-HCT in 67 (35F/32M) SCA children (5-15 year) on CT for abnormal time-averaged mean maximum velocities (TAMMV ≥ 200 cm/s). Seven had a stroke history. We reported that MSD-HCT reduced the highest TAMMVs at 1-and 3-year (p < 0.001) and improved quality of life (QoL) for physical and school functioning. In stroke-free patients, the 3-year stenosis score was lower (p = 0.010). Nevertheless, no significant difference was observed for silent cerebral infarcts (SCI) and cognitive performance. This prompted us to initiate DREPAGREFFE-2 to reevaluate the outcomes at 10 years (September 2022/August 2024) with the same 67 SCA children. No death or stroke occurred in either arm. No rejection or chronic-GvHD arose in the MSD-HCT group (n=32). In the standard-care group (n=35), 16 were on hydroxyurea, and 16 on CT at Year 10, and 3 received haploidentical-HCT. After MSD-HCT, the QoL was better, even for social functioning, and the number of hospitalizations, hospitalized days (p<0.001), and crises (p=0.001) was lower than on standardcare. In stroke-free patients, stenosis (p=0.027) and SCI scores (p=0.041) decreased significantly more after MSD-HCT than on standard-care; working memory (p=0.016) and processing speed (p=0.011) improved significantly after MSD-HCT, but worsened on standard-care. These effects were not previously detected with shorter follow-up. This supports earlier consideration of HCT for SCA children with MSD to preserve neurologic function and QoL for a more productive future.
SN - 0361-8609
SN - 1096-8652
ER -