Aplastic anemia: management of adult patients

The primary therapeutic approach to acquired aplastic anemia (AA) in older adults differs from the primary approach used in children and younger adults because in the former group, the results of allogeneic bone marrow transplantation (BMT) are less favorable. With increasing age of the patients, immunosuppressive therapy with antithymocyte globulin (ATG) and cyclosporine (CsA) constitutes the primary treatment option and may be better than BMT. There are very few clinical clues as to the selection of patients likely to respond to immunosuppression. Repeated ATG/CsA cycles are often used as salvage regimens, but in refractory patients BMT may be the best treatment option, as the prognosis for non-responders is poor without definitive treatment. Conservative therapy such as intense immunosuppression is associated with a high relapse rate but does not impact the survival and overall prognosis. The inability to eliminate autoimmune T cell clones using current therapeutic strategies suggests that prolonged immunosuppressive maintenance therapy may be needed for a substantial proportion of patients. Late clonal complications of conservatively treated patients include evolution to myelodysplasia and paroxysmal nocturnal hemoglobinuria and may develop in 20% of the patients. However, BMT also has several sequelae including an increased frequency of solid tumors. Novel immunosuppressive and immunomodulatory agents and constantly improving results of allogeneic BMT will further improve the survival rate of adult patients with AA.