Allogeneic hematopoietic cell transplantation for aplastic anemia: a single institution experience across 6 decades

Since the introduction of allogeneic bone marrow transplantation (BMT) as a treatment for aplastic anemia (AA), major advances have occurred, such as refinements in conditioning regimens, graft-versus-host disease (GVHD) prophylaxis, high-resolution HLA typing, pretransplant 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 AA over 6 decades at a single BMT center. We highlight key temporal changes in conditioning strategies for related donor transplantation, GVHD prophylaxis, and HLA matching that correspond with improved nonrelapse 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 AA among 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 antithymocyte globulin in conditioning regimens, improve GVHD prevention and management, and expand the use of alternative donors.