Allogeneic Hematopoietic Cell Transplantation in Patients aged 50 years or older with Severe Aplastic Anemia

Highlights

The first global study of HCT for SAA in older patients.

Age does not impact on survival.

Choice of GVHD prophylaxis and performance score impact on outcomes.

Abstract

We report on 499 patients with severe aplastic anemia aged ≥50 years who underwent hematopoietic cell transplantation (HCT) from HLA-matched sibling (n=275), or HLA-matched (8/8) unrelated donors (n=39187) between 2005 and 2016. The median age at HCT was 57.8 years; 16% of patients were 65-77 years old. Multivariable analysis confirmed higher mortality risks for patients with performance score less than 90% (HR 1.41 (1.03-1.92), p=0.03) and after unrelated donor transplantation (HR 1.47 (1-2.16), p=0.05). The 3-year probabilities of survival for patients with performance score 90-100 and less than 90 after HLA-matched sibling transplant were 66% (57-75%) and 57% (47-76%), respectively. The corresponding probabilities after HLA-matched unrelated donor transplantation were 57% (48-67%) and 48% (36-59%). Age at transplantation was not associated with survival but grade II-IV acute GVHD risks were higher for patients aged 65 years or older (sHR 1.7 (1.07-2.72), p=0.026). Chronic GVHD was lower with GVHD prophylaxis regimens calcineurin inhibitor (CNI) + methotrexate (sHR 0.52 (0.33-0.81), p=0.004) and CNI alone or other agents (sHR 0.27 (0.14-0.53), p<0.001) compared to CNI + mycophenolate. While 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.