IMPACT OF ABATACEPT-BASED GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS ON TRANSPLANT OUTCOMES IN CHILDREN WITH HEMOGLOBINOPATHIES: A SYSTEMATIC REVIEW
- 1Faculdade Ciências Médicas de Minas Gerais (FCM-MG), Belo Horizonte, MG, Brazil
- 2Instituto de Oncologia Ciências Médicas (IONCM-CMMG), Belo Horizonte, MG, Brazil
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.
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BibTeX
@article{viana2026impact,
title = {{IMPACT OF ABATACEPT-BASED GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS ON TRANSPLANT OUTCOMES IN CHILDREN WITH HEMOGLOBINOPATHIES: A SYSTEMATIC REVIEW}},
author = {Pedro Henrique Rodrigues VIANA and Lucas De Barros ANASTÁCIO},
journal = {Hematology, Transfusion and Cell Therapy},
year = {2026},
month = oct,
doi = {10.1016/j.htct.2026.106877},
url = {https://doi.org/10.1016/j.htct.2026.106877},
note = {CC BY},
}
RIS
TY - JOUR
TI - IMPACT OF ABATACEPT-BASED GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS ON TRANSPLANT OUTCOMES IN CHILDREN WITH HEMOGLOBINOPATHIES: A SYSTEMATIC REVIEW
AU - Pedro Henrique Rodrigues VIANA
AU - Lucas De Barros ANASTÁCIO
PY - 2026
DA - 2026/10/23
JO - Hematology, Transfusion and Cell Therapy
DO - 10.1016/j.htct.2026.106877
UR - https://doi.org/10.1016/j.htct.2026.106877
AB - 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.
SN - 2531-1379
SN - 2531-1387
ER -