THE SIGNIFICANCE OF MODERN TREATMENT MODALITIES: IMMUNOSUPPRESSIVE THERAPY AND BONE MARROW TRANSPLANTATION IN APLASTIC ANEMIA
Keywords:
Keywords: Aplastic anemia, immunosuppressive therapy, hematopoietic stem cell transplantation, antithymocyte globulin, eltrombopag, haploidentical transplantation, bone marrow failure, cyclosporineAbstract
ABSTRACT
Aplastic anemia (AA) is a rare but life-threatening hematological disorder
characterized by bone marrow failure, resulting in pancytopenia and significant
morbidity and mortality. The management of AA has undergone remarkable
transformation over recent decades, with two principal therapeutic modalities—
immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell
transplantation (HSCT)—forming the cornerstone of modern treatment algorithms.
This comprehensive review examines the current state and clinical significance of these
approaches in the management of AA. The pathophysiological basis of acquired AA
involves a cytotoxic T-cell-mediated autoimmune attack against hematopoietic stem
cells, providing the rationale for IST. Standard IST combining antithymocyte globulin
(ATG) with cyclosporine achieves overall response rates of 60-70%, while the addition
of thrombopoietin receptor agonists such as eltrombopag has revolutionized outcomes,
improving both response rates and response quality. Concurrently, advances in
transplantation technology—including improved HLA typing, reduced-intensity
conditioning regimens, post-transplant cyclophosphamide for GVHD prophylaxis, and
expansion of donor options to include matched unrelated and haploidentical donors—
have made HSCT safer and more accessible. The choice between IST and HSCT
depends on multiple factors including patient age, comorbidities, donor availability,
and disease severity. Emerging evidence supports upfront alternative donor
transplantation in selected populations, challenging traditional treatment paradigms.
This review synthesizes current evidence from recent clinical trials, meta-analyses, and
international guidelines, providing a comprehensive overview of modern AA
management and future therapeutic directions.
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