Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia Journal Article
| Authors: | 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.; Gómez-Almaguer, D.; Vicent, M. G.; Hashem, H.; Kamble, R. T.; Kasow, K. A.; Lazarus, H. M.; Mathews, V.; Orchard, P. J.; Pulsipher, M.; Ringden, O.; Schultz, K.; Teira, P.; Woolfrey, A. E.; Saldaña, B. D.; Savani, B.; Winiarski, J.; Yared, J.; Weisdorf, D. J.; Antin, J. H.; Eapen, M. |
| Article Title: | Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia |
| 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. © 2019 American Society of Hematology. All rights reserved. |
| Keywords: | adolescent; adult; child; controlled study; survival rate; human cell; major clinical study; overall survival; busulfan; fludarabine; methotrexate; allele; cyclophosphamide; allogenic bone marrow transplantation; retrospective study; age; acute graft versus host disease; chronic graft versus host disease; hla matching; whole body radiation; graft versus host reaction; transplantation conditioning; tacrolimus; cyclosporine; graft recipient; thymocyte antibody; aplastic anemia; drug choice; clinical outcome; mycophenolic acid; human; male; female; priority journal; article; matched unrelated donor; matched sibling donor |
| Journal Title: | Blood Advances |
| Volume: | 3 |
| Issue: | 20 |
| ISSN: | 2473-9529 |
| Publisher: | American Society of Hematology |
| Date Published: | 2019-10-22 |
| Start Page: | 3123 |
| End Page: | 3131 |
| Language: | English |
| DOI: | 10.1182/bloodadvances.2019000722 |
| PUBMED: | 31648332 |
| PROVIDER: | scopus |
| PMCID: | PMC6849938 |
| DOI/URL: | |
| Notes: | Source: Scopus |
