Stenger EO, Watkins B, Rogowski K, et al. Abatacept GVHD Prophylaxis in Unrelated Hematopoietic Cell Transplantation for Pediatric Bone Marrow Failure. Blood Advances. 2023; (doi: 10.1182/bloodadvances.2022008545).

Researchers found that adding four doses of abatacept to the standard two-drug graft-versus-host disease (GVHD) prophylaxis in unrelated donor (URD) hematopoietic cell transplantation (HCT) among patients with pediatric bone marrow failure was safe, with encouraging efficacy. The single-arm, single-center Phase I trial included 10 patients receiving reduced intensity or non-myeloablative conditioning underwent URD HCT. Nine patients had successful engraftment; however, there was one primary graft rejection due to an inadequate cell dose. One patient with concurrent cytomegalovirus viremia experienced secondary graft rejection. There were two deaths unrelated to use of abatacept. One patient developed probable post-transplant lymphoproliferative disease and was treated with rituximab and immune suppression withdrawal. There were no cases of severe acute or chronic GVHD. Eight patients had discontinued systemic immunosuppression by 1 year. Abatacept did not appear to affect immune reconstitution, and the retention of naïve relative to effector memory CD4+ T cells was compared with an earlier trial demonstrating abatacept's efficacy in preventing GVHD following URD HCT for malignant diseases (Aba2 trial).

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Tags: GVHD, transplantation, Research, Transplant, Graft versus host disease, cell therapy, grafts, chronic GVHD, graft-versus-host disease, graft-vs-host disease, hematopoietic transplantation, hematopoietic cell transplantation