Autologous transplant in the treatment of severe aplastic anemia – A case report

Abstract

The initial use of immunosuppressive therapy (IST) in severe aplastic anemia (sAA) or reapplication of IST-centered methods following disease relapse is successful only in well-selected patients. The potential treatment by autologous stem cell (SC) transplant in sAA is still an innovative/pioneering therapeutic approach. To our best knowledge, this is the second published case of autologous SC transplant in sAA. The aim of this work was to optimize mobilization and timing for SC harvesting – using our own controlled-rate cryopreservation – with higher CD34 +/CD90 + subset yield and recovery in order to obtain complete and long-term hematopoietic reconstitution following autologous SC transplant. We report a 35 year-old sAA male patient who initially underwent IST using rabbit ATG and Cyclosporine A (CsA). He was supportive transfusion dependent for the whole period of IST-phase. After the second IST-cycle, polymorphonuclear (PMN) cell count increase (>2.0 × 10 9/L) was observed, when SC mobilization, two large volume leukapheresis procedures and following autologous transplant were performed. The yields of harvested CD34 + and CD34 +/CD90 + cells were 5.75 × 10 6/kgbm and 1.7 × 10 6/kgbm, respectively. The quantity of applied CD34 + and CD34 +/CD90 + cells in autologous SC transplant were 5.45 × 10 6/kgbm (7-AAD CD34 + viability = 95.42%) and 1.63 × 10 6/kgbm (7-AAD CD34 + /CD90 + viability = 95.42%), respectively. Hematopoietic reconstitution registered due to second month after autologous SC transplant and he is 24 months in complete medullar, hematological and clinical remission, with normal cytogenetic status – applying only continuous CsA therapy. The results obtained strongly confirm that in sAA, with no allogeneic SC donor, autologous transplant can result in a successful clinical outcome. We suggest that CD34 +/CD90 + subset count in peripheral blood and/or cell-harvest could be more valuable predictive factor than total CD34 + quantity of optimized collection-timing and superior treatment efficacy of autologous SC transplant in sAA.