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Blood Adv. 2019 Oct 22;3(20):3123-3131. doi: 10.1182/bloodadvances.2019000722.

Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia.

Author information

1
Department of Blood and Marrow Transplant and Cellular Therapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL.
2
Division of Biostatistics and.
3
Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI.
4
The Ottawa Hospital Blood & Marrow Transplant Program, University of Ottawa, Ottawa, ON, Canada.
5
Children's Center for Cancer and Blood Diseases, Children's Hospital of Los Angeles, Los Angeles, CA.
6
Division of Pediatric Hematology/Oncology, Children's Mercy Hospital, Kansas City, MO.
7
King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
8
Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Evanston, IL.
9
Department of Medicine, University of Miami, Miami, FL.
10
Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.
11
Hospital Niño Jesus, Madrid, Spain.
12
Division of Pediatric Blood and Marrow Transplantation, University of California San Francisco Medical Center, San Francisco, CA.
13
Clinical Genetics Branch, National Cancer Institute, Rockville, MD.
14
Department of Medicine, Oregon Health & Science University, Portland, OR.
15
Division of Experimental Medicine, Department of Medicine, Imperial College London, London, United Kingdom.
16
Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS.
17
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
18
Department of Hematology, Christian Medical College Hospital, Vellore, India.
19
Department of Medicine, Weill Cornell Medical College, New York, NY.
20
Hospital Universitario José E. González, Universidad Autónoma de Nuevo León, Monterrey, Mexico.
21
Division of Hematology and Oncology, Nationwide Children's Hospital, Columbus, OH.
22
Department of Medicine. Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX.
23
Department of Pediatrics, University of North Carolina Hospitals, Chapel Hill, NC.
24
Department of Medicine, Case Western Reserve University, Cleveland, OH.
25
Department of Medicine, University of Minnesota, Minneapolis, MN.
26
Division of Therapeutic Immunology, Karolinska Institutet, Stockholm, Sweden.
27
British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada.
28
Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada.
29
Fred Hutchinson Cancer Research Center, Seattle, WA.
30
Children's National Medical Center, Washington, DC.
31
Vanderbilt University Medical Center, Nashville, TN.
32
Greenebaum Cancer Center, University of Maryland, Baltimore, MD; and.
33
Stem Cell Transplantation, Dana-Farber Cancer Institute, Boston, MA.

Abstract

Allogeneic bone marrow transplantation (BMT) is curative therapy for the treatment of patients with severe aplastic anemia (SAA). However, several conditioning regimens can be used for BMT. We evaluated transplant conditioning regimens for BMT in SAA after HLA-matched sibling and unrelated donor BMT. For recipients of HLA-matched sibling donor transplantation (n = 955), fludarabine (Flu)/cyclophosphamide (Cy)/antithymocyte globulin (ATG) or Cy/ATG led to the best survival. The 5-year probabilities of survival with Flu/Cy/ATG, Cy/ATG, Cy ± Flu, and busulfan/Cy were 91%, 91%, 80%, and 84%, respectively (P = .001). For recipients of 8/8 and 7/8 HLA allele-matched unrelated donor transplantation (n = 409), there were no differences in survival between regimens. The 5-year probabilities of survival with Cy/ATG/total body irradiation 200 cGy, Flu/Cy/ATG/total body irradiation 200 cGy, Flu/Cy/ATG, and Cy/ATG were 77%, 80%, 75%, and 72%, respectively (P = .61). Rabbit-derived ATG compared with equine-derived ATG was associated with a lower risk of grade II to IV acute graft-versus-host disease (GVHD) (hazard ratio [HR], 0.39; P < .001) but not chronic GVHD. Independent of conditioning regimen, survival was lower in patients aged >30 years after HLA-matched sibling (HR, 2.74; P < .001) or unrelated donor (HR, 1.98; P = .001) transplantation. These data support Flu/Cy/ATG and Cy/ATG as optimal regimens for HLA-matched sibling BMT. Although survival after an unrelated donor BMT did not differ between regimens, use of rabbit-derived ATG may be preferred because of lower risks of acute GVHD.

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