What is the role of immunosuppressive therapy (IST) in the treatment of severe and very severe aplastic anemia (SAA or VSAA)?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Immunosuppressive therapy using antithymocyte globulin (ATG) and cyclosporine is used as first-line therapy [89] for patients with severe or very severe aplastic anemia (SAA or VSAA, respectively) who are older than 50 years (35-50 years in presence of comorbidities) and as second-line therapy in younger patients with SAA or VSAA if a human leukocyte antigen (HLA)–matched sibling donor is not available. Immunosuppressive therapy is also recommended in patients with nonsevere aplastic anemia who are transfusion dependent. [5] Immunosuppressive therapy is associated with an overall response rate of 60-80% and a 5-year survival rate of 75% in most reports, but event-free survival rates are in the range of 35-50%.


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