Blood Advances (Oct 2019)
Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia
- Nelli Bejanyan,
- Soyoung Kim,
- Kyle M. Hebert,
- Natasha Kekre,
- Hisham Abdel-Azim,
- Ibrahim Ahmed,
- Mahmoud Aljurf,
- Sherif M. Badawy,
- Amer Beitinjaneh,
- Jaap Jan Boelens,
- Miguel Angel Diaz,
- Christopher C. Dvorak,
- Shahinaz Gadalla,
- James Gajewski,
- Robert Peter Gale,
- Siddhartha Ganguly,
- Andrew R. Gennery,
- Biju George,
- Usama Gergis,
- David Gómez-Almaguer,
- Marta Gonzalez Vicent,
- Hasan Hashem,
- Rammurti T. Kamble,
- Kimberly A. Kasow,
- Hillard M. Lazarus,
- Vikram Mathews,
- Paul J. Orchard,
- Michael Pulsipher,
- Olle Ringden,
- Kirk Schultz,
- Pierre Teira,
- Ann E. Woolfrey,
- Blachy Dávila Saldaña,
- Bipin Savani,
- Jacek Winiarski,
- Jean Yared,
- Daniel J. Weisdorf,
- Joseph H. Antin,
- Mary Eapen
Affiliations
- Nelli Bejanyan
- Department of Blood and Marrow Transplant and Cellular Therapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL;
- Soyoung Kim
- Division of Biostatistics, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI;
- Kyle M. Hebert
- Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI;
- Natasha Kekre
- The Ottawa Hospital Blood & Marrow Transplant Program, University of Ottawa, Ottawa, ON, Canada;
- Hisham Abdel-Azim
- Children's Center for Cancer and Blood Diseases, Children's Hospital of Los Angeles, Los Angeles, CA;
- Ibrahim Ahmed
- Division of Pediatric Hematology/Oncology, Children's Mercy Hospital, Kansas City, MO;
- Mahmoud Aljurf
- King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia;
- Sherif M. Badawy
- Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Evanston, IL;
- Amer Beitinjaneh
- Department of Medicine, University of Miami, Miami, FL;
- Jaap Jan Boelens
- Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY;
- Miguel Angel Diaz
- Hospital Niño Jesus, Madrid, Spain;
- Christopher C. Dvorak
- Division of Pediatric Blood and Marrow Transplantation, University of California San Francisco Medical Center, San Francisco, CA;
- Shahinaz Gadalla
- Clinical Genetics Branch, National Cancer Institute, Rockville, MD;
- James Gajewski
- Department of Medicine, Oregon Health & Science University, Portland, OR;
- Robert Peter Gale
- Division of Experimental Medicine, Department of Medicine, Imperial College London, London, United Kingdom;
- Siddhartha Ganguly
- Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS;
- Andrew R. Gennery
- Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom;
- Biju George
- Department of Hematology, Christian Medical College Hospital, Vellore, India;
- Usama Gergis
- Department of Medicine, Weill Cornell Medical College, New York, NY;
- David Gómez-Almaguer
- Hospital Universitario José E. González, Universidad Autónoma de Nuevo León, Monterrey, Mexico;
- Marta Gonzalez Vicent
- Hospital Niño Jesus, Madrid, Spain;
- Hasan Hashem
- Division of Hematology and Oncology, Nationwide Children's Hospital, Columbus, OH;
- Rammurti T. Kamble
- Department of Medicine. Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX;
- Kimberly A. Kasow
- Department of Pediatrics, University of North Carolina Hospitals, Chapel Hill, NC;
- Hillard M. Lazarus
- Department of Medicine, Case Western Reserve University, Cleveland, OH;
- Vikram Mathews
- Department of Hematology, Christian Medical College Hospital, Vellore, India;
- Paul J. Orchard
- Department of Medicine, University of Minnesota, Minneapolis, MN;
- Michael Pulsipher
- Children's Center for Cancer and Blood Diseases, Children's Hospital of Los Angeles, Los Angeles, CA;
- Olle Ringden
- Division of Therapeutic Immunology, Karolinska Institutet, Stockholm, Sweden;
- Kirk Schultz
- British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada;
- Pierre Teira
- Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada;
- Ann E. Woolfrey
- Fred Hutchinson Cancer Research Center, Seattle, WA;
- Blachy Dávila Saldaña
- Children's National Medical Center, Washington, DC;
- Bipin Savani
- Vanderbilt University Medical Center, Nashville, TN;
- Jacek Winiarski
- Division of Therapeutic Immunology, Karolinska Institutet, Stockholm, Sweden;
- Jean Yared
- Greenebaum Cancer Center, University of Maryland, Baltimore, MD
- Daniel J. Weisdorf
- Department of Medicine, University of Minnesota, Minneapolis, MN;
- Joseph H. Antin
- Stem Cell Transplantation, Dana-Farber Cancer Institute, Boston, MA.
- Mary Eapen
- Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI;; Mary Eapen, Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, 9200 W Wisconsin Ave, Suite C5500, Milwaukee, WI 53226;
- Journal volume & issue
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Vol. 3,
no. 20
pp. 3123 – 3131
Abstract
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 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.
