再生不良性貧血の現在の治療戦略

Aplastic anemia is a bone marrow failure syndrome characterized by bone marrow hypoplasia and pancytopenia, often involving a T cell-mediated autoimmune mechanism. Treatment modalities aimed at hematopoietic recovery include immunosuppressive therapy, thrombopoietin receptor agonists (TPO-RA), and allogeneic hematopoietic stem cell transplantation (allo-SCT). The combination of TPO-RA with conventional immunosuppressive therapy using anti-thymocyte globulin and cyclosporine has become a standard treatment option. In addition to improved outcomes with allo-SCT from HLA-matched sibling donors, favorable results have recently been reported for cord blood transplantation and haploidentical allo-SCT, expanding the range of effective treatment options. As treatment outcomes and life expectancy have improved, the impact of relapse and clonal evolution following immunosuppressive therapy, as well as secondary malignancies and late complications after allo-SCT, has become increasingly significant, representing important challenges for the future.