We conducted a retrospective analysis of outcomes for pediatric patients (<=22 years) at two centers who received a 7/8 MMUD HCT for hematologic malignancies and received GVHD prophylaxis with abatacept (4 doses, 10 mg/kg per dose, on days –1, + 5, + 14, and + 28) + CNI/MTX between January 2015 and December 2021. The analysis included pediatric patients transplanted at Children’s Healthcare of Atlanta and Boston Children’s Hospital. To compare these abatacept-treated patients to the most rigorous control, we included a comparison group from both centers who received an 8/8 HLA matched HCT for a hematologic malignancy, but who received CNI/MTX without abatacept (or other GVHD prophylaxis). Patients were excluded if they were on the ABA2 trial, not in remission at the time of HCT, received a prior HCT, or received GVHD prophylaxis other than CNI/MTX + abatacept.
Univariate analyses were performed to describe and compare outcomes between the groups. Grade II-IV aGVHD, grade III-IV aGVHD, moderate to severe chronic graft-versus-host disease (cGVHD), TRM and relapse are described as cumulative incidence estimates. Competing risks were relapse and death for the GVHD outcomes, relapse for TRM, and death in the absence of relapse for the relapse endpoint. Gray’s test was used to compare outcomes between the groups. OS, DFS, and grade III-IV acute GVHD-free, severe cGVHD-free, relapse-free survival (GRFS) are described as Kaplan-Meier survival probability estimates. The log-rank test was used to compare curves between the groups. All of the time-to-event endpoints are defined as the time from HCT until the event, the competing event, or until their last follow-up, whichever occurred first. Acute GVHD is analyzed at 180 days, while all other endpoints are analyzed at 1 year.
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- Source: https://www.nature.com/articles/s41409-023-02034-z