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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

Title
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
Authors
Kang, Hyoung JinHong, Kyung TaekLee, Ji WonKim, HyeryPark, Kyung DukShin, Hee YoungLee, Soo HyunYoo, Keon HeeSung, Ki WoongKoo, Hong HoeLee, Jae WookChung, Nak GyunCho, BinKim, Hack KiKoh, Kyung NamIm, Ho JoonSeo, Jong JinJung, Hyun JooPark, Jun EunLee, Young HoLim, Young TakLim, Yeon JungKim, Sun YoungYoo, Eun SunRyu, Kyung HaLee, Jae HeePark, Jeong-APark, Sang KyuAhn, Hyo SeopKorean Soc Pediat Hematology-Oncol
Ewha Authors
유경하유은선
SCOPUS Author ID
유경하scopus; 유은선scopus
Issue Date
2016
Journal Title
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
ISSN
1083-8791JCR Link1523-6536JCR Link
Citation
vol. 22, no. 8, pp. 1455 - 1459
Keywords
Severe aplastic anemiaUnrelated donorFludarabineCyclophosphamideThymoglobulinAntithymocyte globulin (ATG)
Publisher
ELSEVIER SCIENCE INC
Indexed
SCI; SCIE; SCOPUS WOS scopus
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. (C) 2016 American Society for Blood and Marrow Transplantation.
DOI
10.1016/j.bbmt.2016.04.003
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의학전문대학원 > 의학과 > Journal papers
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