Aplastic anemia - diagnosis and treatment of this complex disease

Abstract:

Aplastic anemia (AA) is heterogeneous disease characterized by hypocellular or acellular bone marrow and bi-cytopenia or pancytopenia and not just anemia in peripheral blood in the most cases. Aplastic anemia can be inherited or acquired. The principle mechanism leading to bone marrow failure and secondary cytopenia in the most cases of acquired AA is immune-mediated destruction of hematopoietic stem cells. Severe AA can be treated effectively with immunosuppressive therapy or allogeneic hematopoietic stem cell transplantation in selected patients, and eltrombopag, an orally agonist of thrombopoietin receptors, has been introduced in the AA therapy recently. One third of patients have disease that is refractory to immunosuppression, with persistent, severe pancytopenia, bleeding diathesis, infections and frequent transfusions of blood products.

Key words:
allogeneic hematopoietic stem cell transplantation; aplastic anemia; immunosuppressive therapy; eltrombopag