First-Line Treatment Options for Severe Aplastic Anemia: Transplantation vs. Immunosuppressive Therapy

Severe aplastic anemia (SAA) is a life-threatening blood disorder characterized by bone marrow failure and a significant reduction in the production of all blood cell types. Effective treatment depends heavily on patient-specific factors, particularly age and the availability of a suitable donor. Medical guidelines emphasize two primary therapeutic pathways: hematopoietic stem cell transplantation (HSCT) for eligible patients with matched donors, and immunosuppressive therapy (IST) for those without.

When Stem Cell Transplantation Is the Preferred Option

For younger patients—typically under the age of 45—stem cell transplantation is considered the first-line treatment when a fully matched sibling donor is available. This approach offers the best chance for long-term cure, as it replaces the defective bone marrow with healthy stem cells capable of normal blood cell production.

Finding the Right Donor Match

The ideal scenario involves a human leukocyte antigen (HLA)-matched sibling, which significantly reduces the risk of graft-versus-host disease (GVHD) and transplant-related complications. When such a match is confirmed through genetic testing, physicians generally recommend proceeding with allogeneic HSCT as early as possible to maximize outcomes.

Immunosuppressive Therapy for Patients Without a Matched Donor

In cases where no suitable donor is available, immunosuppressive therapy becomes the cornerstone of treatment. The standard regimen includes a combination of cyclosporine and anti-thymocyte globulin (ATG). This dual approach works by suppressing the overactive immune system that attacks the bone marrow, thereby allowing residual stem cells to recover and resume blood cell production.

Managing Very Severe Cases: Haploidentical Transplants

For patients diagnosed with very severe aplastic anemia—often presenting with critical cytopenias and high infection risks—alternative donor options may be explored. One promising option is haploidentical stem cell transplantation, which uses partially matched donors such as a parent or half-matched sibling. Advances in conditioning regimens and post-transplant care have improved the safety and efficacy of this approach, making it a viable rescue strategy for high-risk individuals.

Ultimately, treatment decisions must be personalized, integrating clinical severity, patient age, donor availability, and institutional expertise. Early diagnosis and timely intervention remain key to improving survival and quality of life in patients with severe aplastic anemia.