{
  "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.",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 41,
  "authors": [
    {
      "name": "Francoise Bernaudin"
    },
    {
      "name": "Suzanne Verlhac"
    },
    {
      "name": "Elisabeth Ducros‐miralles"
    },
    {
      "name": "Cécile Arnaud"
    },
    {
      "name": "Isabelle Genty"
    },
    {
      "name": "Marie Petras"
    },
    {
      "name": "Catherine Paillard"
    },
    {
      "name": "Gonzalo de Luna"
    },
    {
      "name": "Benoit Meunier"
    },
    {
      "name": "Isabelle Thuret"
    },
    {
      "name": "Annie Kamdem"
    },
    {
      "name": "Corinne Pondarré"
    },
    {
      "name": "Giovanna Cannas"
    },
    {
      "name": "Christelle Chantalat‐auger"
    },
    {
      "name": "Nadia Firah"
    },
    {
      "name": "Sophie Pertuisel"
    },
    {
      "name": "Laure Joseph"
    },
    {
      "name": "Florence Missud"
    },
    {
      "name": "Mariane de Montalembert"
    },
    {
      "name": "Corinne Guitton"
    },
    {
      "name": "Claire Pluchart"
    },
    {
      "name": "Valentine Brousse"
    },
    {
      "name": "Monique Elmaleh"
    },
    {
      "name": "Vincent Gajdos"
    },
    {
      "name": "Emmanuella Leveillé"
    },
    {
      "name": "Ekaterina Belozertseva"
    },
    {
      "name": "Mabel Gaba"
    },
    {
      "name": "Catherine Poirot"
    },
    {
      "name": "Virginie Barraud‐lange"
    },
    {
      "name": "Philippe Chadebech"
    },
    {
      "name": "France Pirenne"
    },
    {
      "name": "Myriam Bernaudin"
    },
    {
      "name": "Bénédicte Neven"
    },
    {
      "name": "Pierre Frange"
    },
    {
      "name": "Marie Angoso"
    },
    {
      "name": "Karima Yakouben"
    },
    {
      "name": "Bénédicte Bruno"
    },
    {
      "name": "Nathalie Dhédin"
    },
    {
      "name": "Jean‐hugues Dalle"
    },
    {
      "name": "Regis Peffault Delatour"
    },
    {
      "name": "Camille Jung"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/54yejvc28rbwk.bib",
  "cited_by": 4,
  "doi": "10.1002/ajh.70336",
  "fulltext_url": null,
  "id": "54yejvc28rbwk",
  "issns": [
    "0361-8609",
    "1096-8652"
  ],
  "landing_url": "https://normandie-univ.hal.science/hal-05769867v1",
  "language": "en",
  "licence": "CC-BY-NC-ND-4.0",
  "licence_label": "CC BY-NC-ND 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "hal",
      "0.sdv"
    ],
    [
      "openalex",
      "2716|Hemoglobinopathies and Related Disorders"
    ]
  ],
  "openalex": "W7162750079",
  "pdf_url": "https://normandie-univ.hal.science/hal-05769867/document",
  "pmcid": null,
  "pmid": null,
  "published": "2026-05-28",
  "publisher": "Wiley",
  "ris": "https://research.draconitic.com/paper/54yejvc28rbwk.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:17:43Z",
      "native_id": "hal-05769867",
      "source": "hal",
      "url": "https://normandie-univ.hal.science/hal-05769867v1"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2716|Hemoglobinopathies and Related Disorders",
      "subfield": "human-genetics"
    }
  ],
  "title": "Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial",
  "title_text": "Better 10‐Year Cerebrovascular Outcome After Transplant Than on Standard‐Care in Sickle Cell Anemia: DREPAGREFFE Trial",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:17:43Z",
  "url": "https://research.draconitic.com/paper/54yejvc28rbwk",
  "venue": "American Journal of Hematology",
  "versions": []
}