Abstract
We report on 499 patients with severe aplastic anemia aged >/= 50years who underwent hematopoietic cell transplantation (HCT) from HLA-matched sibling (n=275, 55%) or HLA-matched (8/8) unrelated donors (n=187, 37%) between 2005 and 2016. The median age at HCT was 57.8 years; 16% of patients were 65 to 77years old. Multivariable analysis confirmed higher mortality risks for patients with performance score less than 90% (hazard ratio [HR], 1.41; 95% confidence interval [CI], 1.03 to 1.92; P=.03) and after unrelated donor transplantation (HR, 1.47; 95% CI, 1 to 2.16; P=.05). The 3-year probabilities of survival for patients with performance scores of 90 to 100 and less than 90 after HLA-matched sibling transplant were 66% (range, 57% to 75%) and 57% (range, 47% to 76%), respectively. The corresponding probabilities after HLA-matched unrelated donor transplantation were 57% (range, 48% to 67%) and 48% (range, 36% to 59%). Age at transplantation was not associated with survival, but grades II to IV acute graft-versus-host disease (GVHD) risks were higher for patients aged 65years or older (subdistribution HR [sHR], 1.7; 95% confidence interval, 1.07 to 2.72; P=.026). Chronic GVHD was lower with the GVHD prophylaxis regimens calcineurin inhibitor (CNI)+methotrexate (sHR, .52; 95% CI, .33 to .81; P=.004) and CNI alone or with other agents (sHR, .27; 95% CI, .14 to .53; P < .001) compared with CNI+mycophenolate. Although donor availability is modifiable only to a limited extent, choice of GVHD prophylaxis and selection of patients with good performance scores are key for improved outcomes.
