Abatacept for Graft-Versus-Host Disease Prevention in Pediatric Patients

By Patrick Daly - Last Updated: April 26, 2022

A study, presented by Sharmila Raghunandan, DO, MPH, at the Transplantation & Cellular Therapy 2022 Tandem Meetings, observed “encouragingly” positive results in reducing severe acute graft-versus-host disease (aGVHD), transplant-related mortality (TRM), and relapse, and improving event-free survival (EFS) and overall survival (OS) in high-risk pediatric patients receiving abatacept for GVHD prophylaxis after mismatched unrelated donor (MMURD) hematopoietic cell transplant (HCT), as compared to a contemporaneous HLA matched cohort without abatacept.

However, the study’s authors acknowledged that, “while abatacept reduced severe AGVHD, CGVHD remains a concern with the 4-dose regimen.”

The trial retrospectively analyzed 26 pediatric patients from a single center who received 7/8 MMUD HCT with abatacept for GVHD prophylaxis, as well as a calcineurin inhibition (CNI) and methotrexate (MTX). A comparison cohort was formed of patients who received an 8/8 HLA matched unrelated donor (MUD) with only CNI and MTX.

According to the authors, the incidence of grade II-IV and grade III-IV aGVHD in the abatacept group was 55.5% and 7.7%, respectively, compared to 57.5% and 18.1% in the MUD group (p = 0.647 and p = 0.275). Additional comparative outcomes included:

  • Moderate to severe cGVHD at one year was 56.4% in the abatacept group compared to 23.4% in the MUD group (p = 0.058).
  • Severe aGVHD relapse-free survival (SGRFS) at day 180 was 76.5% in the abatacept group and 65.5% in the MUD group (p = 0.36).
  • One-year TRM was 3.9% and 20.7% in the abatacept and MUD group, respectively (P = 0.087).
  • EFS and OS at one year were 80.1% and 88.1%, respectively with abatacept, compared to 65.5% and 69% with MUD (p = 0.25 and p = 0.10).
  • Relapse at one year was 16.7% with abatacept compared to 13.8% with MUD (p = 0.837).

Overall, the authors concluded that abatacept may be effective for the high-risk pediatric patient population undergoing MMURD HCT for hematological malignancies, though they noted that “extended dosing should be considered to mitigate the risk of cGVHD.” In closing, they called for future studies to further investigate the efficacy of abatacept for prevention of severe aGVHD.

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