Steroids for Aplastic Anemia
Corticosteroids alone have no role as primary therapy for aplastic anemia and should not be used as monotherapy for this condition. 1
Primary Treatment Framework
The standard first-line treatment for severe aplastic anemia depends on age and donor availability, not corticosteroids:
For Patients Under 50 Years
- Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is first-line therapy if an HLA-matched sibling donor is available. 1
- The conditioning regimen includes Cyclophosphamide-ATG (Cy-ATG), with Cyclophosphamide 200 mg/kg total dose (days -5 to -2) and ATG 11.25-15.00 mg/kg total dose (days -5 to -3 or -2). 1
For Patients Without Matched Donors or Over 50 Years
- Immunosuppressive therapy with horse antithymocyte globulin (ATG) plus cyclosporine is the standard first-line treatment. 1, 2, 3
- The standard ATG dosing is 40 mg/kg per day for 4 days, combined with cyclosporine 10-12 mg/kg per day for 6 months (adjusted for blood levels). 2
- Lower dose ATG (25 mg/kg/day × 4 days) has shown similar efficacy in resource-constrained settings with overall response rates of 77% at 6 months. 4
Limited Role of Corticosteroids
Corticosteroids are only used as a short adjunctive course during ATG administration, not as primary therapy:
- A brief course of methylprednisolone (1 mg/day for approximately 2 weeks) is given alongside ATG to manage serum sickness and infusion reactions. 2
- This represents supportive care during immunosuppression, not therapeutic intervention for the aplastic anemia itself. 2
Treatment Outcomes and Response Assessment
Response to ATG plus cyclosporine occurs in approximately 60% of patients at 3 months, with excellent long-term survival in responders:
- Response rates: 60% at 3 months, 61% at 6 months, and 58% at 1 year. 2
- Overall actuarial survival at 7 years is 55%, but survival among responders reaches 86% at 5 years. 2
- Early hematologic response (reticulocyte count or platelet count >50 × 10³/μL at 3 months) predicts 90% survival at 5 years. 2
- There were no deaths among responders more than 3 years after treatment. 2
Second-Line Treatment for Refractory Disease
For patients under 50 years who fail immunosuppression, proceed to allo-HSCT with haploidentical donor (HID), matched unrelated donor (MUD), or cord blood transplant (CBT). 1
- For patients 50-60 years with ECOG ≤2 who failed immunosuppression, MSD or MUD transplantation is recommended. 1
- Relapse after ATG/cyclosporine is common but does not influence survival, and severe pancytopenia usually does not recur. 2
Critical Pitfalls to Avoid
Do not confuse aplastic anemia with myelodysplastic syndromes (MDS): The evidence provided discusses immunosuppression for hypoplastic MDS, which is a different entity. 5 In MDS with marrow hypoplasia, immunomodulatory treatment similar to aplastic anemia may be offered with limited success. 5
Do not delay treatment: Longer intervals between diagnosis and initiation of IST (>6 months) are associated with worse response rates and survival. 6, 4
Do not use rabbit ATG as first-line: Horse ATG has been shown to be superior to rabbit ATG for first-line treatment of severe aplastic anemia. 6