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Diseases

Abatacept Prevents Severe Acute Graft-Versus-Host Disease in Pediatric Bone Marrow Failure Syndromes

Strategic Insights

— 2 min read

Research published in Pediatric Blood & Cancer has found that the medication Abatacept prevents severe acute graft-versus-host disease (GVHD) without increasing the incidence of graft failure in pediatric patients with bone marrow failure syndromes. A study conducted by researchers at Cincinnati Children's Hospital Medical Center involved 26 patients who received Abatacept as part of their hematopoietic stem cell transplant (HSCT) treatment, and compared their outcomes to a historical cohort of 21 patients who did not receive Abatacept. The results showed that none of the patients in the Abatacept group experienced severe acute GVHD, compared to 14% in the historical cohort, with all patients in the Abatacept group successfully engrafting.

Key Takeaways:

  • Abatacept prevented severe acute graft-versus-host disease (GVHD) in pediatric patients with bone marrow failure syndromes.
  • None of the 26 patients in the Abatacept group experienced severe (Grade III-IV) acute GVHD, compared to 14% in the historical cohort (CI 0% vs. 14%, p = 0.05).
  • All patients in the Abatacept group successfully engrafted, with one patient experiencing poor graft function.
  • The rates of chronic GVHD were comparable between the historical and Abatacept groups (28.6% vs. 19.2%, p = 0.51).
  • The overall survival (OS) (95.2% vs. 96.0%, p = 0.3) and graft failure-free overall survival (GFS) at 1 year (64.3% vs. 65.3%, p = 0.5) were similar in the Abatacept group compared to the historical cohort.
  • Abatacept was well tolerated in patients with BMFS, preventing severe acute GVHD without increasing the incidence of graft failure.

Statistics:

  • 26 patients received Abatacept as part of their HSCT treatment.
  • 21 patients in the historical cohort did not receive Abatacept.
  • The rates of chronic GVHD were 28.6% in the historical cohort and 19.2% in the Abatacept group.
  • The overall survival (OS) was 95.2% in the Abatacept group and 96.0% in the historical cohort.
  • The graft failure-free overall survival (GFS) at 1 year was 64.3% in the Abatacept group and 65.3% in the historical cohort.
  • Abatacept was administered to patients at various dosages.

Sources:

  • Abatacept Prevents Severe Acute Graft-Versus-Host Disease Without Increasing Graft Failure Risk in Pediatric Bone Marrow Failure Syndromes. Pediatric Blood & Cancer, 2025.
  • Stella M. Davies, Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States.
  • Zahra Hudda, Adam Lane, Deborah E. Schiff, Eric J. Anderson, Parinda A. Mehta, Kasiani C. Myers, and Nicholas J. Gloude, authors of the research.
  • Hematology Week, October 13, 2025, p 302.

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