Science Highlights
Published on July 15, 2025
Allogeneic transplant outcomes with PTCy and donor type in SAA
by Blood Advances
Montoro J, Eikema D-J, Piepenbroek B, et al. Donor Impact on Allogeneic Transplant Outcomes with PTCy for Severe Aplastic Anemia: A Study of the SAAWP EBMT. Bone Marrow Transplantation. 2025; (doi: 10.1038/s41409-025-02633-y).
Registry data suggest post-transplant cyclophosphamide (PTCy) is safe and effective for patients with severe aplastic anemia (SAA) who undergo stem cell transplantation (SCT), especially with HLA-matched sibling (MSD) and unrelated donors (UDs). Investigators examined data from the European Society for Bone and Marrow Transplantation on SAA patients who had SCT during 2010-2022 and received PTCy as prevention against graft-versus-host disease (GVHD). A total of 209 patients in the analysis had haploidentical donors, while 70 had MSDs and 69 had UDs. Researchers compared outcomes across donor categories, noting two-year chronic GVHD rates ranging between 13% and 14% and 2-year extensive chronic GVHD rates ranging between 2% and 6%. The safety profile was favorable, with 2-year overall survival (OS) rates of 92% for MSDs and 81% for UDs and GVHD- and relapse-free survival (GRFS) rates of 70% and 66%, respectively. Comparatively, OS and GRFS rates were 66% and 54%, respectively, among SAA patients with haploidentical donors—lower rates that were attributed to 2-year non-relapse mortality of 24%, versus 7% for MSDs and 10% for UDs.
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