| Summary: | Aplastic anemia (AA) is a rare disorder characterized by suppression of bone marrow function. It can develop as the
result of congenital marrow disease and chemical exposure;
however, most cases are idiopathic.1 Treatment with immunosuppressive therapy (IST) for patients who do not have an
human leukocyte antigen (HLA)-compatible donor relies on
the evidence that a deregulated immune system drives T
lymphocytes to cytokine-mediated destruction of their own
hematopoietic stem cells.1 The majority of these patients
respond well to up-front administration of IST, including antithymocyte globulin (ATG) and cyclosporine (CsA), which is
successful in around 80%.2 Unfortunately, ATG and CsA can
lead to clonal disorders, in particular myelodysplastic syndrome (MDS) and paroxysmal nocturnal hemoglobinuria
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