Acquired severe aplastic anemia (SAA) is a rare hematologic disease associated with significant morbidity and mortality. Immune destruction of hemopoietic stem cells, plays an important role in the pathogenesis, as shown by successful treatment with immunosuppressive agents (IST), leading to transfusion independence, or complete recovery of peripheral blood counts, in a proportion of patients. Growth factors, can be combined with IST, and may improve response rates, as recently shown with thrombopoietin analogs. Anabolic steroids may still play a role in combination with IST. The problem with IST is failure to respond and the development of late clonal disorders. Bone marrow transplantation (BMT) , is the other therapeutic option: a matched sibling donor remains the best choice. For patients lacking a matched family donor, unrelated donors can be readily found, although mostly for patients of caucasian origin. Other BMT options include unrelated cord blood or mismatched family donors. Acute and chronic graft versus host disease remain important complications of BMT. Patients age is a strong predictor of outcome for both IST and BMT, and must be considered when designing therapeutic strategies. Early diagnosis and treatment, as well as long term monitoring, remain crucial steps for successful treatment of SAA.
- aplastic anemia