Aplastic anemia (AA) is an autoimmune disease that reduces the amount of blood cells of all types produced by the bone marrow. This pancytopenia causes a host of symptoms, including fatigue, reduced immunity and dizziness. The condition can be hereditary or develop after a trigger, such as exposure to cytotoxins. However, many cases of AA are idiopathic.
AA can become chronic and severe. In such cases allogeneic hematopoietic stem cell transplant may be a first-line treatment. Therapeutic options also include immunosuppressive agents like anti-thymocyte globulin, steroids and chemotherapy. Targeted agents like eltrombopag are more recent developments.
Patients with AA are at risk for later developing paroxysmal nocturnal hemoglobinuria, myelodysplastic syndromes and acute leukemias. A greater understanding of the dysregulation of immune mechanisms that leads to AA is needed to lead to improvements in the field.