Treatment Approaches for Aplastic Anemia: From Immunosuppression to Stem Cell Transplantation

Aplastic anemia is typically managed through a stratified treatment approach, tailored to the severity of the condition. This personalized strategy ensures that patients receive the most effective and appropriate care based on their specific clinical presentation.

Managing Non-Severe Aplastic Anemia

For patients diagnosed with non-severe aplastic anemia, immunosuppressive therapy plays a central role. The most commonly prescribed medication is cyclosporine, an oral immunosuppressant that helps modulate the immune system's attack on bone marrow. In addition to cyclosporine, androgen therapy—such as stanozolol or danazol—may be introduced to stimulate red blood cell production and improve hematopoietic function.

Some treatment regimens also incorporate traditional herbal formulations like Compound Zhaofan Pills, which are believed to support bone marrow recovery, although scientific validation is still ongoing. Another adjunctive option is levamisole, an immunomodulatory agent that may enhance immune regulation and complement standard therapies.

Treatment Strategies for Severe and Very Severe Cases

In pediatric patients with severe or very severe aplastic anemia, hematopoietic stem cell transplantation (HSCT) is considered the first-line curative option, especially when a matched donor is available. HSCT offers the potential for long-term remission by replacing damaged bone marrow with healthy stem cells.

Alternative Immunosuppressive Regimens

When a suitable donor is unavailable or families express concerns about transplantation, combination immunosuppressive therapy becomes a viable alternative. This typically involves antithymocyte globulin (ATG) or antilymphocyte globulin (ALG) administered alongside cyclosporine. This dual approach effectively suppresses the autoimmune destruction of bone marrow and has demonstrated significant response rates in clinical settings.

Essential Supportive Care Measures

Beyond primary treatments, comprehensive supportive care is critical for managing symptoms and preventing complications. One cornerstone of this care is component transfusion therapy.

Red blood cell transfusions are recommended when hemoglobin levels fall below 60 g/L to alleviate symptoms of anemia such as fatigue, shortness of breath, and pallor. Similarly, platelet transfusions are indicated when platelet counts drop below 10×10⁹/L to reduce the risk of spontaneous bleeding. In cases of active hemorrhage, prompt platelet infusion is essential to control bleeding and stabilize the patient.

Preventing and Managing Infections

Due to compromised bone marrow function, patients often have neutropenia, making them highly susceptible to infections. Therefore, aggressive infection prevention and early intervention are crucial. Prophylactic antibiotics, antifungals, and vigilant monitoring for signs of sepsis form key components of care. Any suspected infection should be treated promptly with broad-spectrum antimicrobials while awaiting culture results.

Addressing Iron Overload from Chronic Transfusions

Frequent blood transfusions, while life-saving, can lead to iron overload—a condition where excess iron accumulates in organs such as the liver, heart, and endocrine glands. This can result in organ dysfunction over time. To mitigate this risk, chelation therapy using agents like deferasirox or deferoxamine is initiated when iron burden reaches critical levels. Regular monitoring of serum ferritin and organ function helps guide the timing and intensity of iron removal strategies.

Overall, the management of aplastic anemia requires a multidisciplinary approach involving hematologists, transplant specialists, infectious disease experts, and supportive care teams. With advances in immunosuppressive protocols, stem cell transplantation techniques, and supportive therapies, long-term outcomes continue to improve for both children and adults affected by this rare but serious disorder.