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 pathogenesis, as shown by successful treatment with immunosuppressive agents, leading to transfusion independence or complete recovery of peripheral blood counts in a proportion of patients. Growth factors can be combined
with immunosuppressive therapy (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 graftversus-host disease remain important complications of BMT. Patient 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. (Blood. 2017;129(11):1428-1436)

BLOOD, 16 MARCH 2017 x VOLUME 129, NUMBER 11