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High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.
|Title||High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.|
|Publication Type||Journal Article|
|Year of Publication||2010|
|Authors||Brodsky RA, Chen AR, Dorr D, Fuchs EJ, Huff CA, Luznik L, Smith DB, Matsui WH, Goodman SN, Ambinder RF, Jones RJ|
|Date Published||2010 Mar 18|
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder that can be treated with bone marrow transplantation, immunosuppressive therapy, and high-dose cyclophosphamide. Here, we report long-term follow-up on 67 SAA patients (44 treatment-naive and 23 refractory) treated with high-dose cyclophosphamide. At 10 years, the overall actuarial survival was 88%, the response rate was 71% with the majority being complete, and the actuarial event-free survival was 58% in 44 treatment-naive SAA patients. Patients with refractory SAA fared less well after high-dose cyclophosphamide therapy; at 10 years, overall actuarial survival, response, and actuarial event-free survival rates were 62%, 48%, and 27%, respectively. High-dose cyclophosphamide is highly effective therapy for severe aplastic anemia. Large randomized controlled trials will be necessary to establish how results of high-dose cyclophosphamide compare with either bone marrow transplantation or standard immunosuppressive regimens, such as antithymocyte globulin and cyclosporine.