What is the treatment for aplastic anemia?

Treatment of Aplastic Anemia

The first-line treatment for aplastic anemia is immunosuppressive therapy with antithymocyte globulin (ATG) and cyclosporine A for patients without a matched sibling donor, while HLA-identical sibling hematopoietic stem cell transplantation (HSCT) is recommended for patients younger than 40 years who have a suitable donor. 1

Diagnostic Approach

Before initiating treatment, confirm the diagnosis with:

• Complete blood count showing pancytopenia
• Bone marrow aspirate and biopsy demonstrating hypocellularity
• Exclusion of other causes of bone marrow failure

Treatment Algorithm

First-line Treatment Options:

1. For patients <40 years with an HLA-identical sibling donor:

   • Allogeneic HSCT is the preferred treatment
   • Conditioning regimen: cyclophosphamide and ATG
   • Stem cell source: bone marrow (preferred over peripheral blood)
   • GVHD prophylaxis: cyclosporine A and methotrexate 1

2. For patients without a matched sibling donor or >40 years:

   • Immunosuppressive therapy (IST) with:
     • ATG (preferably horse ATG)
     • Cyclosporine A
     • Consider adding eltrombopag 2

For Severe Aplastic Anemia with FDA-approved indication:

• Eltrombopag is indicated for adult patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy 3
• Dosing: Start at 50 mg daily (25 mg daily for patients of East Asian ancestry or with hepatic impairment)
• Monitor platelet counts and adjust dose accordingly
• Monitor liver function tests regularly

Supportive Care Measures

• Transfusion support for anemia and thrombocytopenia
  • Maintain platelet counts above 10-20 × 10⁹/L to prevent bleeding
  • Transfuse packed red blood cells for symptomatic anemia
• Infection prevention and prompt treatment of infections
• Growth factors may be considered in severely neutropenic patients with infections, though evidence for continuous use is limited

Treatment Response and Follow-up

• Monitor blood counts weekly until stable, then monthly
• Evaluate response to immunosuppressive therapy at 3-6 months
• For non-responders to initial IST, consider:
  • Second course of ATG (preferably rabbit ATG if horse ATG was used initially)
  • Alternative donor HSCT
  • Clinical trials

Special Considerations

• Patients with relapsed or refractory disease after IST may be candidates for HSCT with alternative donors
• Carefully select patients for HSCT, as the procedure carries significant morbidity
• For patients unsuited for HSCT, best supportive care or palliative systemic treatment may be reasonable options 4

Common Pitfalls to Avoid

1. Delaying treatment in severe aplastic anemia
2. Failure to rule out inherited bone marrow failure syndromes before initiating treatment
3. Not considering HSCT early in the disease course for eligible patients
4. Inadequate supportive care during immunosuppressive therapy
5. Overlooking the development of clonal evolution to myelodysplastic syndrome or acute leukemia during follow-up

The treatment approach for aplastic anemia has significantly improved survival rates, with more than 75% of patients becoming long-term survivors when diagnosed and treated appropriately 1. The choice between HSCT and immunosuppressive therapy should be based on patient age, donor availability, disease severity, and comorbidities.