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

TitleHigh-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.
Publication TypeJournal Article
Year of Publication2010
AuthorsBrodsky RA, Chen AR, Dorr D, Fuchs EJ, Huff CA, Luznik L, Smith DB, Matsui WH, Goodman SN, Ambinder RF, Jones RJ
JournalBlood
Volume115
Issue11
Pagination2136-41
Date Published2010 Mar 18
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.

DOI10.1089/scd.2009.0326
Alternate JournalBlood