High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.

Division of Hematology,Department of Medicine, Johns Hopkins University School ofMedicine, 720 Rutland Ave, Ross Bldg, Rm 1025, Baltimore, MD 21205, USA.
Blood (Impact Factor: 9.78). 12/2009; 115(11):2136-41. DOI: 10.1182/blood-2009-06-225375
Source: PubMed

ABSTRACT 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.

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