Management and Treatment of Aplastic Anemia
Summary
Aplastic anaemia is a life-threatening bone marrow failure syndrome characterised by pancytopenia and markedly reduced marrow cellularity. Management strategies centre on two principal approaches: immunosuppressive therapy to halt immune-mediated destruction of haematopoietic stem and progenitor cells, and haematopoietic stem cell transplantation to restore marrow function. First-line immunosuppression typically combines anti-thymocyte globulin with ciclosporin, often supplemented by thrombopoietin receptor agonists such as eltrombopag to enhance multilineage recovery. For younger patients with a matched sibling donor, allogeneic transplantation offers the best chance of durable remission, while alternative donor platforms—including haploidentical transplantation—have expanded curative options for patients lacking a fully matched graft. Supportive care with transfusions, infection prophylaxis and growth factors remains integral throughout treatment, with close monitoring to detect clonal evolution or progression to myeloid malignancies. Recent advances include refined conditioning regimens to reduce toxicity, novel cellular therapies targeting the bone marrow niche, and the integration of real-time genomic and immunological profiling to guide personalised interventions. Globally, improved donor registries and access to novel agents have narrowed outcome disparities, emphasising the potential for tailored strategies that balance efficacy, toxicity and resource availability.
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Management and Treatment of Aplastic Anemia publication trend
The graph below shows the total number of articles in management and treatment of aplastic anemia across all publications each year (not limited to Nature Index journals).
Technical terms
Haematopoietic stem cell transplantation (HSCT): Infusion of donor stem cells to repopulate a patient’s bone marrow.
Immunosuppressive therapy (IST): Treatment combining agents that inhibit immune-mediated destruction of marrow cells.
Anti-thymocyte globulin (ATG): Polyclonal antibodies targeting T cells to prevent immune attack on progenitor cells.
Ciclosporin (CyA): Calcineurin inhibitor used to suppress T cell activity in aplastic anaemia.
Eltrombopag: Thrombopoietin receptor agonist that stimulates platelet and progenitor cell production.
Haploidentical transplant: HSCT using a donor who shares only one HLA haplotype with the recipient.
Ferroptosis: Iron-dependent form of regulated cell death contributing to haematopoietic stem cell loss.
References
- Unbiased Single‐Cell Sequencing of Hematopoietic and Immune Cells from Aplastic Anemia Reveals the Contributors of Hematopoiesis Failure and Dysfunctional Immune Regulation. Advanced Science (2023).
- Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study. Leukemia (2023).
- Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant. Journal of Hematology & Oncology (2017).
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