1. Improved Outcome of a Reduced Toxicity-Fludarabine, Cyclophosphamide, plus Antithymocyte Globulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia: Comparison of 2 Multicenter Prospective Studies.
- Author
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Kang, Hyoung Jin, Hong, Kyung Taek, Lee, Ji Won, Kim, Hyery, Park, Kyung Duk, Shin, Hee Young, Lee, Soo Hyun, Yoo, Keon Hee, Sung, Ki Woong, Koo, Hong Hoe, Lee, Jae Wook, Chung, Nak Gyun, Cho, Bin, Kim, Hack Ki, Koh, Kyung Nam, Im, Ho Joon, Seo, Jong Jin, Jung, Hyun Joo, Park, Jun Eun, and Lee, Young Ho
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FLUDARABINE , *CYCLOPHOSPHAMIDE , *THYMOCYTES , *GLOBULINS , *APLASTIC anemia , *HEMATOPOIETIC stem cell transplantation - Abstract
Hematopoietic stem cell transplantation (HSCT) is a curative therapy for severe aplastic anemia (SAA); however, the optimal conditioning regimen for HSCT with an unrelated donor has not yet been defined. A previous study using a fludarabine (FLU), cyclophosphamide (Cy), and antithymocyte globulin (ATG) conditioning regimen (study A: 50 mg/kg Cy once daily i.v. on days −9, −8, −7, and −6; 30 mg/m 2 FLU once daily i.v. on days −5, −4, −3, and −2; and 2.5 mg/kg of ATG once daily i.v. on days −3, −2, and −1) demonstrated successful engraftment (100%) but had a high treatment-related mortality rate (32.1%). Therefore, given that Cy is more toxic than FLU, we performed a new phase II prospective study with a reduced-toxicity regimen (study B: 60 mg/kg Cy once daily i.v. on days −8 and −7; 40 mg/m 2 FLU once daily i.v. on days −6, −5, −4, −3, and −2; and 2.5 mg/kg ATG once daily i.v. on 3 days). Fifty-seven patients were enrolled in studies A (n = 28) and B (n = 29), and donor type hematologic recovery was achieved in all patients in both studies. The overall survival (OS) and event-free survival (EFS) rates of patients in study B was markedly improved compared with those in study A (OS: 96.7% versus 67.9%, respectively, P = .004; EFS: 93.3% versus 64.3%, respectively, P = .008). These data show that a reduced-toxicity conditioning regimen with FLU, Cy, and ATG may be an optimal regimen for SAA patients receiving unrelated donor HSCT. [ABSTRACT FROM AUTHOR]
- Published
- 2016
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