Treatment of Severe Aplastic Anemia

First-Line Treatment Algorithm by Age and Donor Availability

For patients under 50 years with severe aplastic anemia and an HLA-matched sibling donor, allogeneic hematopoietic stem cell transplantation (allo-HSCT) should be offered as first-line therapy; for those without a matched donor or over 50 years, immunosuppressive therapy with horse antithymocyte globulin (ATG) plus cyclosporine is the standard first-line treatment, according to the Journal of Hematology and Oncology guidelines 1
The American Society of Hematology recommends proceeding directly to allo-HSCT as first-line therapy for severe or very severe aplastic anemia (SAA/vSAA) in patients under 50 years with a matched sibling donor 1
The conditioning regimen for HLA-matched sibling transplantation 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), as recommended by the Journal of Hematology and Oncology 2, 3

The American Society of Hematology recommends proceeding to allo-HSCT with haploidentical donor (HID), matched unrelated donor (MUD), or cord blood transplant (CBT) for patients under 50 years who failed immunosuppression, with a modified conditioning regimen including mBuCyFluATG 1, 2, 3
For patients 50-60 years with ECOG ≤2 who failed immunosuppression, the American Society of Hematology recommends MSD or MUD transplantation 1