Since the introduction of allogeneic bone marrow transplantation (BMT) for aplastic anemia, major advances have included refinements in conditioning regimens, graft-versus-host disease (GVHD) prophylaxis, high-resolution human leukocyte antigen (HLA) typing, pre-transplant transfusion practices, and general supportive care. We present a comprehensive retrospective single-center cohort study of 607 children and adults who underwent allogeneic transplantation for aplastic anemia over six decades at a single BMT center. We highlight key temporal changes in conditioning for related donor transplants, GVHD prophylaxis, and HLA matching that correspond with improved non-relapse mortality, reduced GVHD rates, and better overall survival among HLA-matched related and unrelated donor transplants. This work provides a historical perspective on the evolution of BMT for aplastic anemia for HLA-matched related and unrelated donor recipients and identifies persistent barriers to curative therapy, including patient age and donor availability. Further studies are needed to clarify the role of anti-thymocyte globulin in conditioning regimens, improve GVHD prevention and management, and expand use of alternative donors.
Olivieri et al. (2026) studied this question.