Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial
Year: 2026
Type: article
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 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.
Source: American Journal of Hematology
DOI: 10.1002/ajh.70336
Authors: Francoise Bernaudin, Suzanne Verlhac, Elisabeth Ducros‐Miralles, Cécile Arnaud, Isabelle Genty, Marie Petras, Catherine Paillard, Gonzalo De Luna, Benoit Meunier, Isabelle Thuret, Annie Kamdem, Corinne Pondarré, Giovanna Cannas, Christelle Chantalat‐Auger, Nadia Firah, Sophie Pertuisel, Laure Joseph, Florence Missud, Mariane de Montalembert, Corinne Guitton, Claire Pluchart, Valentine Brousse, Monique Elmaleh, Vincent Gajdos, Emmanuella Leveillé, Ekaterina Belozertseva, Mabel Gaba, Catherine Poirot, Virginie Barraud‐Lange, Philippe Chadebech, France Pirenne, Myriam Bernaudin, Bénédicte Neven, Pierre Frange, Marie Angoso, Karima Yakouben, Bénédicte Bruno, Nathalie Dhédin, Jean‐Hugues Dalle, Regis Peffault Delatour, Camille Jung
Institutions: Université Paris-Est Créteil, Hôpital Intercommunal de Créteil, Hôpital Robert-Debré, Centre Hospitalier Universitaire Pointe-à-Pitre, Hôpital d'Hautepierre, Hôpitaux Universitaires Henri-Mondor, Hôpital de la Conception, Hôpital de la Timone, Hôpital Edouard Herriot, Bicêtre Hospital, Centre Hospitalier De Pau, Université de Rennes, Hôpital Necker-Enfants Malades, Assistance Publique – Hôpitaux de Paris, Hôpital Saint-Louis, Inserm, Centre National de la Recherche Scientifique, Normandie Université, Cyceron, Imagerie et Stratégies Thérapeutiques pour les Cancers et Tissus cérébraux, Université de Caen Normandie, Centre Hospitalier Universitaire de Bordeaux, Université Paris Cité, Centre Hospitalier Universitaire de Lille
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $5,450
Funders: Agence de la Biomédecine, Pfizer France
Awards: G2180335425, G803186151
OpenAlex ID: W7162750079