High Overall and GVHD-Free Survival in Patients with Aplastic Anemia Receiving in vivo T-cell Depletion Transplants and Long-Term Complications

We report a single-center experience of allogeneic hematopoietic stem cell transplant for patients with severe aplastic anemia over 13 years of age. Fifty-eight patients were included, and in vivo T-cell depletion was used in all cases. Fifty-one (88%) received alemtuzumab and 7 (12%) were given rabbit anti-thymocyte globulin. The median follow-up period was 6 years (range: 0-13.5). Data was collected retrospectively and updated in April 2019. The median age was 31 years (range: 18-67). Forty (69%) recipients received grafts from related donors and 18 (31%) received them from unrelated donors. Peripheral blood grafts were infused in 12 (20.7%) patients. Five-year overall survival (OS) was 80.7%. Five-year graft-versus-host disease (GVHD)/rejection-free survival was 56%. Eight (13.8%) patients experienced graft failure. The cumulative incidence of grade II-IV acute GVHD at day 100 was 14% and that of chronic GVHD at 1 year was 7%. The selection of unrelated donors and the use of peripheral blood grafts were not significant risk factors for clinically relevant GVHD or for lower OS. Recipients older than 40 years showed significantly worse OS, as observed from the results of univariate analysis. T-cell depletion in severe aplastic anemia shows low rates of GVHD and high OS, but older patients remain a group with higher risk of mortality. Long-term complications were mainly autoimmune in character.