Clinical management of aplastic anemia

Abstract

Acquired aplastic anemia is a potentially fatal bone marrow failure disorder that is characterized by pancytopenia and a hypocellular bone marrow. Hematopoietic stem-cell transplantation or bone marrow transplantation (BMT) is the treatment of choice for young patients who have a matched sibling donor. Immunosuppression with either anti-thymocyte globulin and cyclosporine or high-dose cyclophosphamide is an effective therapy for patients who are not suitable BMT candidates owing to age or lack of a suitable donor. Results of BMT from unrelated and mismatched donors are improving, but presently this treatment option is best reserved for those patients who do not respond, relapse or develop secondary clonal disorders following immunosuppressive therapy. Efforts are currently underway to both improve immunosuppressive regimens and to expand the application of BMT.

Figure 1. Results of high-dose cyclophosphamide therapy for severe aplastic anemia

(A) Overall survival after high-dose cyclophosphamide therapy for 44 treatment-naive patients (upper line) and 23 patients refractory to prior immunosuppressive therapy (lower line), p = 0.03 (log rank test). (B) Failure-free survival after high-dose cyclophosphamide therapy for 44 treatment-naive patients (upper line) and 23 patients refractory to prior immunosuppressive therapy (lower line), p = 0.07 (log rank test). This research was originally published in [76]. © The American Society of Hematology.