Abatacept for Prophylaxis of Acute and Chronic Graft-Versus-Host-Disease in Children Undergoing Allogeneic Hematopoietic Stem Cell Transplantation-A Systematic Review and Meta-Analysis of Cohort Studies
In brief
Abatacept regimens cut severe acute graft-versus-host disease by 86% in children
Across 15 cohort studies, abatacept-containing regimens were linked to 69% fewer cases of moderate-to-severe acute graft-versus-host disease and 86% fewer severe cases after pediatric stem cell transplant. They did not reduce chronic disease overall, though extended treatment was associated with fewer cases; retrospective designs and varied study populations limit confidence in the findings.
- Journal
- Pediatric blood & cancer (Q1)
- Published
- 10 October 2026
- Study design
- Systematic review of cohort studies
- Evidence level
- Level 2, Moderate (CEBM 2a)
- Authors
- Matthijs D Kruizinga, Anne B Verbeek, Caroline A Lindemans, Emilie P Buddingh
- PMID
- 42858118
- DOI
- 10.1002/1545-5017.70746
Why clinicians should know about it
- Picked for Hematology (paper of the day, 13 October 2026): Abatacept prophylaxis for GvHD in pediatric HSCT
Abstract
BACKGROUND: Graft-versus-host disease (GvHD) remains one of the most significant complications of hematopoietic stem cell transplantation (HSCT). Abatacept, a selective T-cell costimulation modulator, is a relatively novel prophylactic agent, though pediatric data remain limited. PROCEDURE: A systematic review and meta-analysis was conducted to evaluate the efficacy and safety of abatacept for GvHD prophylaxis in pediatric HSCT. RESULTS: Fifteen cohort studies were identified (eight comparative and seven single-arm). Studies included abatacept as adjunctive agent to traditional regimens. Pooled analyses demonstrated that abatacept-inclusive regimens significantly reduced the risk of Grade II-IV acute GvHD (aGvHD) (risk ratio [RR] 0.31, 95% confidence interval [CI] 0.14-0.65) and severe Grade III-IV aGvHD (RR 0.14, 95% CI 0.05-0.37). Abatacept administration was not associated with decreased moderate-to-severe chronic GvHD incidence, although extended regimens were associated with reduced risk (RR 0.25, 95% CI 0.08-0.78). Abatacept-inclusive GvHD prophylaxis was not associated with compromised immune reconstitution or increased viral reactivations, whereas it was associated with a reduced incidence of bacterial infections (RR 0.39, 95% CI 0.21-0.74) and improved overall survival (RR 1.18, 95% CI 1.03-1.34). CONCLUSIONS: Despite limitations inherent to retrospective study designs and cohort heterogeneity, these findings indicate that abatacept is an efficacious and safe addition to GvHD prophylaxis in pediatric HSCT.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.