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Biol Blood Marrow Transplant. 2016 Aug;22(8):1455-1459. doi: 10.1016/j.bbmt.2016.04.003. Epub 2016 May 8.

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 information

1
Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea.
2
Department of Pediatrics, Sungkyunkwan University School of Medicine, Samsung Medical Center, Seoul, Republic of Korea.
3
Department of Pediatrics, The Catholic University of Korea College of Medicine, Seoul, Republic of Korea.
4
Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Republic of Korea.
5
Department of Pediatrics, Ajou University School of Medicine, Suwon, Republic of Korea.
6
Department of Pediatrics, Hanyang University College of Medicine, Seoul, Republic of Korea.
7
Department of Pediatrics, Pusan National University School of Medicine, Pusan, Republic of Korea.
8
Department of Pediatrics, Chungnam National University College of Medicine, Daejeon, Republic of Korea.
9
Department of Pediatrics, Ewha Woman's University School of Medicine, Seoul, Republic of Korea.
10
Department of Pediatrics, Chung-Ang University Hospital, Seoul, Republic of Korea.
11
Department of Pediatrics, Inje University Haeundae Paik Hospital, Pusan, Republic of Korea.
12
Department of Pediatrics, University of Ulsan College of Medicine, Ulsan University Hospital, Ulsan, Republic of Korea.
13
Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea. Electronic address: hsahn@snu.ac.kr.

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.

KEYWORDS:

Antithymocyte globulin (ATG); Cyclophosphamide; Fludarabine; Severe aplastic anemia; Thymoglobulin; Unrelated donor

PMID:
27090956
DOI:
10.1016/j.bbmt.2016.04.003
[Indexed for MEDLINE]
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