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

• Published in: Blood advances Vol. 3; no. 20; pp. 3123 - 3131
• Main Authors: Bejanyan, Nelli, Kim, Soyoung, Hebert, Kyle M., Kekre, Natasha, Abdel-Azim, Hisham, Ahmed, Ibrahim, Aljurf, Mahmoud, Badawy, Sherif M., Beitinjaneh, Amer, Boelens, Jaap Jan, Diaz, Miguel Angel, Dvorak, Christopher C., Gadalla, Shahinaz, Gajewski, James, Gale, Robert Peter, Ganguly, Siddhartha, Gennery, Andrew R., George, Biju, Gergis, Usama, Gómez-Almaguer, David, Vicent, Marta Gonzalez, Hashem, Hasan, Kamble, Rammurti T., Kasow, Kimberly A., Lazarus, Hillard M., Mathews, Vikram, Orchard, Paul J., Pulsipher, Michael, Ringden, Olle, Schultz, Kirk, Teira, Pierre, Woolfrey, Ann E., Saldaña, Blachy Dávila, Savani, Bipin, Winiarski, Jacek, Yared, Jean, Weisdorf, Daniel J., Antin, Joseph H., Eapen, Mary
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 22-10-2019; American Society of Hematology; Elsevier
• Subjects: Medicin och hälsovetenskap; Transplantation
• ISSN: 2473-9529; 2473-9537; 2473-9537
• DOI: 10.1182/bloodadvances.2019000722

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.
•Flu/Cy/ATG and Cy/ATG regimens offer the best survival for matched-sibling BMT.•Transplantation in patients aged ≥30 years is associated with higher mortality after matched-sibling and unrelated donor BMT.
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