[A new era in the treatment of aplastic anemia]

Abstract

Aplastic anemia (AA) is a rare hematologic disorder characterized by bone marrow hypoplasia and pancytopenia, and is classified into idiopathic and secondary forms. Idiopathic AA is primarily treated with immunosuppressive therapy (IST), thrombopoietin receptor agonists (TPO-RAs), and hematopoietic stem cell transplantation (HSCT). Cyclosporine (CsA) monotherapy is recommended for patients who have mild disease or are moderately transfusion-independent, whereas the combination of anti-thymocyte globulin (ATG) and CsA is the standard treatment for severe disease. In 2023, equine ATG (ATGAM^®) was approved in Japan, expanding the options for IST. TPO-RA options for combination therapy with IST now include romiplostim in addition to eltrombopag, and studies have demonstrated the efficacy of triple combination therapy with ATG, CsA, and a TPO-RA has been demonstrated. In the context of HSCT, HLA-haploidentical transplantation using post-transplant cyclophosphamide is increasingly being considered as an option for patients without an HLA-matched donor due to its improved safety and efficacy. This review provides a comprehensive overview of the latest advances in AA treatment, including novel therapeutic strategies, and discusses future therapeutic directions to further improve patient outcomes.

Keywords: Aplastic anemia; Hematopoietic stem cell transplantation; Immunosuppressive therapy; Thrombopoietin receptor agonists.