TY - JOUR
T1 - High-dose cyclophosphamide for severe aplastic anemia
T2 - Long-term follow-up
AU - Brodsky, Robert A.
AU - Chen, Allen R.
AU - Dorr, Donna
AU - Fuchs, Ephraim J.
AU - Huff, Carol Ann
AU - Luznik, Leo
AU - Smith, B. Douglas
AU - Matsui, William H.
AU - Goodman, Steven N.
AU - Ambinder, Richard F.
AU - Jones, Richard J.
PY - 2010/3/18
Y1 - 2010/3/18
N2 - 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 SAApatients (44 treatment-naiveand23 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%, and27%, 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.
AB - 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 SAApatients (44 treatment-naiveand23 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%, and27%, 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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U2 - 10.1182/blood-2009-06-225375
DO - 10.1182/blood-2009-06-225375
M3 - Article
C2 - 20018919
AN - SCOPUS:77950412952
SN - 0006-4971
VL - 115
SP - 2136
EP - 2141
JO - Blood
JF - Blood
IS - 11
ER -