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-AlmaguerMarta 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*

*Corresponding author for this work

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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 5 955), 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 6 Flu, and busulfan/Cy were 91%, 91%, 80%, and 84%, respectively (P 5 .001). For recipients of 8/8 and 7/8 HLA allele-matched unrelated donor transplantation (n 5 409), 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 5 .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 5 .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.

Original languageEnglish (US)
Pages (from-to)3123-3131
Number of pages9
JournalBlood Advances
Volume3
Issue number20
DOIs
StatePublished - Jan 1 2019

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ASJC Scopus subject areas

  • Hematology

Cite this

Bejanyan, N., Kim, S., Hebert, K. M., Kekre, N., Abdel-Azim, H., Ahmed, I., Aljurf, M., Badawy, S. M., Beitinjaneh, A., Boelens, J. J., Diaz, M. A., Dvorak, C. C., Gadalla, S., Gajewski, J., Gale, R. P., Ganguly, S., Gennery, A. R., George, B., Gergis, U., ... Eapen, M. (2019). Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Advances, 3(20), 3123-3131. https://doi.org/10.1182/bloodadvances.2019000722