Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: <scp>DREPAGREFFE</scp> 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 &lt; 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 &lt; 0.001), and crises ( p = 0.001) was lower than on standard‐care. 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.

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Journal
American Journal of Hematology
Published
2026-05-28
DOI
https://doi.org/10.1002/ajh.70336
Primary Topic
Hemoglobinopathies and Related Disorders
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article
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article

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

Gonzalo De Luna, Catherine Paillard, Sophie Pertuisel, Bénédicte Neven et al.
American Journal of Hematology
Hemoglobinopathies and Related Disorders
article

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

Gonzalo De Luna, Catherine Paillard, Sophie Pertuisel, Bénédicte Neven, I. Genty, Corinne Guitton, Philippe Chadebech, Ekaterina Belozertseva, Jean‐Hugues Dalle, Nadia Firah, Annie Kamdem, Claire Pluchart, Christelle Chantalat‐Auger, Elisabeth Ducros-Miralles, Giovanna Cannas, Karima Yakouben, Camille Jung, Myriam Bernaudin, Marie Angoso, Regis Peffault Delatour, Isabelle Thuret, Emmanuella Leveillé, Monique Elmaleh, Florence Missud, Nathalie Dhédin, Pierre Frange, F Pirenne, Suzanne Verlhac, Dr Virginie Barraud-Lange, Bénedicte Bruno, Vincent Gajdos, Laure Joseph, Marie Petras, Valentine Brousse, Catherine Poirot, Mariane de Montalembert, Mabel Gaba, Cécile Arnaud, Francoise Bernaudin, Benoit Meunier, Corinne Pondarré
article en

Abstract

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 &lt; 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 &lt; 0.001), and crises ( p = 0.001) was lower than on standard‐care. 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.

American Journal of Hematology
Hôpital Necker-Enfants Malades (FR), Centre National de la Recherche Scientifique (FR), Université de Bordeaux (FR), Inserm (FR), Université Paris-Est Créteil (FR), Université Paris Cité (FR), Université de Lille (FR), Saint Louis University Hospital (US), Université Rennes 2 (FR), Assistance Publique Hôpitaux de Marseille (FR), Lille’s Cardiology Hospital (FR), Hôpital de la Conception (FR), Centre Hospitalier De Pau (FR), Centre Hospitalier Universitaire de Reims (FR), Normandie Université (FR), Bordeaux Population Health (FR), Sickle Cell Institute Chhattisgarh, Raipur (IN), Hôpital Edouard Herriot (FR), Hôpital Robert-Debré (FR), Centre Hospitalier Universitaire Pointe-à-Pitre (GP), Hôpital Saint-Louis (FR), Hôpitaux Universitaires de Strasbourg (FR), Hôpital Antoine-Béclère (FR), Cyceron (FR), Hôpital d'Hautepierre (FR), Hôpital Intercommunal de Créteil (FR), Hôpital de la Timone (FR), Bicêtre Hospital (FR), Cell Medica (Switzerland) (CH), Université de Reims Champagne-Ardenne (FR), Université de Caen Normandie (FR)
Openalex Percentile: Top 6%
Hemoglobinopathies and Related Disorders
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