Document Type

Article

Publication Date

10-22-2019

Identifier

DOI: 10.1182/bloodadvances.2019000722; PMCID: PMC6849938

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.

Journal Title

Blood Adv

Volume

3

Issue

20

First Page

3123

Last Page

3131

MeSH Keywords

Adolescent; Adult; Anemia, Aplastic; Bone Marrow Transplantation; Clinical Decision-Making; Disease Management; Graft vs Host Disease; HLA Antigens; Histocompatibility Testing; Humans; Prognosis; Severity of Illness Index; Siblings; Transplantation Conditioning; Transplantation, Homologous; Treatment Outcome; Young Adult

Keywords

Adolescent; Adult; Anemia, Aplastic; Bone Marrow Transplantation; Clinical Decision-Making; Disease Management; Graft vs Host Disease; HLA Antigens; Histocompatibility Testing; Humans; Prognosis; Severity of Illness Index; Siblings; Transplantation Conditioning; Transplantation, Homologous; Treatment Outcome; Young Adult

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