• 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, Jun Eun Park, Young Ho Lee, Young Tak Lim, Yeon Jung Lim, Sun Young Kim, Eun Sun Yoo, Kyung Ha Ryu, Jae Hee Lee, Jeong-A Park, Sang Kyu Park, Hyo Seop Ahn, and Korean Society of Pediatric Hematology-Oncology.
• Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea.
• Biol. Blood Marrow Transplant. 2016 Aug 1; 22 (8): 1455-1459.

AbstractHematopoietic 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.Copyright © 2016 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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