Science Highlights
Published on December 23, 2025
Long-term outcomes of post-transplant cyclophosphamide for SAA
DeZern A, Zahurak M, Cavin T, et al. Long-Term Follow-Up of the Baltimore Experience With Hematopoietic Cell Transplantation for Severe Aplastic Anemia Using Post-Transplant Cyclophosphamide. Transplantation and Cellular Therapy. 2025; (doi: 10.1016/j.jtct.2025.10.021).
Contrary to conventional practice recommending against upfront hematopoietic cell transplantation (HCT) for severe aplastic anemia (SSA), except in the case of young recipients with matched sibling donors, researchers now say that HCT is appropriate for a broader range of patients when paired with post-transplant cyclophosphamide (PTCy). The conclusion draws from the Baltimore experience, named for a diverse cohort of 111 SSA patients with treatment-naive or relapreduced-intensitysed/refractory disease receiving care at Johns Hopkins. The sample population included children and adults and both related HLA-haplo and unrelated donors. Participants underwent bone marrow transplantation using a reduced-intensity conditioning regimen and GVHD prophylaxis with PTCy platform. Investigators reported rapid hematopoietic reconstitution with low levels of morbidity and mortality across the sample, with 3-year overall survival of 92% and graft failure-free survival of 80% at 2 years and 79% at 3 years. GVHD incidence was low. Notably, endpoints were similar between pediatric and adult patients up to age 60 years, and donor type did not influence outcomes. Most patients no longer required immunosuppressive therapy for GVHD by 6 months and returned to premorbid functional status.
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