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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
- 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
- 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; Lee, Young Ho; Lim, Young Tak; Lim, Yeon Jung; Kim, Sun Young; Yoo, Eun Sun; Ryu, Kyung Ha; Lee, Jae Hee; Park, Jeong-A; Park, Sang Kyu; Ahn, Hyo Seop; Korean Soc Pediat Hematology-Oncol
- Ewha Authors
- 유경하; 유은선
- SCOPUS Author ID
- 유경하; 유은선
- Issue Date
- Journal Title
- BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- 1083-8791; 1523-6536
- vol. 22, no. 8, pp. 1455 - 1459
- Severe aplastic anemia; Unrelated donor; Fludarabine; Cyclophosphamide; Thymoglobulin; Antithymocyte globulin (ATG)
- ELSEVIER SCIENCE INC
- SCI; SCIE; SCOPUS
- 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. (C) 2016 American Society for Blood and Marrow Transplantation.
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