Aplastic Anemia: Drug Therapy

Therapeutic target

Restoration of bone marrow function

Therapy recommendations

  • First-line method: allogeneic stem cell transplantation (in which blood stem cells are transferred from a donor to a recipient, i.e., donor and recipient are not the same person in this case); however, it depends on the presence of an HLA-identical donor.
  • Immunosuppressants, these are indicated for:
    • Non-severe aplastic anemia with substitution requirements.
    • Severe/very severe aplastic anemia in patients > 40 years of age.
    • Severe/very severe aplastic anemia in patients < 40 years of age who lack a histocompatible donor or have other reasons not to undergo stem cell transplantation
  • See also under “Further therapy“.

Immunosuppressants

Immunosuppressants suppress the body’s immune system in an attempt to alleviate disease progression and prevent disease progression. Antithymocyte globulin, ciclosporin (cyclosporin A), and prednisolone are the main drugs used for aplastic anemia.