Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8430844 | Biology of Blood and Marrow Transplantation | 2016 | 5 Pages |
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 â9, â8, â7, and â6; 30 mg/m2 FLU once daily i.v. on days â5, â4, â3, and â2; and 2.5 mg/kg of ATG once daily i.v. on days â3, â2, and â1) 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 â8 and â7; 40 mg/m2 FLU once daily i.v. on days â6, â5, â4, â3, and â2; 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.
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Authors
Hyoung Jin Kang, Kyung Taek Hong, Ji Won Lee, Hyery Kim, Kyung Duk Park, Hee Young Shin, Soo Hyun Lee, Keon Hee Yoo, Ki Woong Sung, Hong Hoe Koo, Jae Wook Lee, Nak Gyun Chung, Bin Cho, Hack Ki Kim, Kyung Nam Koh, Ho Joon Im, Jong Jin Seo, Hyun Joo Jung,