{
  "abstract": "Introduction: Sickle cell disease and transfusion-dependent β-thalassemia are major grounds for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children. While this procedure has curative potential, graft-versus-host disease (GVHD) limits its success. Abatacept (through its inhibitory action on T-cell costimulation via the CD80/CD86–CD28 axis), may improve GVHD incidence, overall survival, graft rejection, and immune-mediated toxicity. Objective: This systematic review aimed to critically appraise the impact of abatacept-containing prophylactic regimens on transplant outcomes in pediatric patients with hemoglobinopathies undergoing allo-HSCT. Methods: This systematic review was conducted in accordance with PRISMA guidelines, using PubMed as the sole database. The search strategy combined MeSH terms and free-text terms across four conceptual blocks: abatacept (and its synonyms, including CTLA4-Ig), graft-versus-host disease prophylaxis, hematopoietic stem cell transplantation, hemoglobinopathies (encompassing sickle cell disease and beta-thalassemia), and pediatric population, connected by Boolean operators (AND/OR), with an additional NOT operator applied to exclude animal studies lacking a human component. Studies published between 2020 and 2026 were considered eligible. Publications within the date range, human subject studies, and pediatric populations were included. Exclusion criteria included adult-only studies or studies with unextractable or qualitative pediatric subgroup outcome data. Separately and blindly, studies were chosen. The selection process, included studies, and main findings were characterized by a qualitative synthesis. Results: The literature search identified seven studies, six of which met the eligibility criteria and were included in the qualitative synthesis. Collectively, 222 patients were analyzed, of whom 160 received abatacept-containing GVHD prophylaxis; 134 of these had a confirmed diagnosis of hemoglobinopathy. The included studies evaluated abatacept-based GVHD prophylaxis in children undergoing allo-HSCT from matched related, matched unrelated, and haploidentical donors. The addition of abatacept was consistently associated with a reduction in severe (grade III–IV) acute GVHD. Its effect on chronic GVHD was more heterogeneous: extended-duration regimens showed significant reductions, whereas shorter regimens were associated with persistently high incidence (up to 57%). Overall survival remained high (87-100%) among intervention groups, exceeding that of control groups in three comparative studies. Reductions in endothelial injury syndromes and transplant-related mortality were also observed. Viral reactivation (cytomegalovirus, Epstein-Barr virus) occurred in both groups without a consistent statistically significant difference between them, representing the principal safety concern identified. Conclusion: Adding abatacept to GVHD prophylaxis may improve GVHD incidence, overall survival, graft rejection, and immune-mediated toxicity. Abatacept reduced severe acute GVHD safely and effectively, with more consistent benefit against chronic GVHD with prolonged-duration regimens. However, observational studies and single-arm cohorts with heterogeneous protocols and small sample sizes provide most of the evidence. Insufficient data on quality of life, fertility, and long-term outcomes necessitates adequately powered randomized controlled trials.",
  "abstract_shown": true,
  "affiliations": [
    "Faculdade Ciências Médicas de Minas Gerais (FCM-MG), Belo Horizonte, MG, Brazil",
    "Instituto de Oncologia Ciências Médicas (IONCM-CMMG), Belo Horizonte, MG, Brazil"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 2,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Pedro Henrique Rodrigues VIANA"
    },
    {
      "affs": [
        1
      ],
      "name": "Lucas De Barros ANASTÁCIO"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/ezw8fnr12wa0a.bib",
  "cited_by": 0,
  "doi": "10.1016/j.htct.2026.106877",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926006243",
  "id": "ezw8fnr12wa0a",
  "issns": [
    "2531-1379",
    "2531-1387"
  ],
  "landing_url": "https://doi.org/10.1016/j.htct.2026.106877",
  "language": "en",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2720|Hematopoietic Stem Cell Transplantation"
    ]
  ],
  "openalex": "W7223137359",
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/ezw8fnr12wa0a.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:26Z",
      "native_id": "92230687e97e476da0a4e05dd1f80684",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.htct.2026.106877"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC633-647.5",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "IMPACT OF ABATACEPT-BASED GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS ON TRANSPLANT OUTCOMES IN CHILDREN WITH HEMOGLOBINOPATHIES: A SYSTEMATIC REVIEW",
  "title_text": "IMPACT OF ABATACEPT-BASED GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS ON TRANSPLANT OUTCOMES IN CHILDREN WITH HEMOGLOBINOPATHIES: A SYSTEMATIC REVIEW",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:26Z",
  "url": "https://research.draconitic.com/paper/ezw8fnr12wa0a",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}