Fludarabine-based reduced intensity regimen for matched related donor hematopoietic stem cell transplantation in acquired severe aplastic anemia

Abstract Different conditioning regimens have been evaluated in matched-related donor allogeneic hematopoietic stem cell transplantation (allo-HSCT) for acquired severe aplastic anemia (SAA) with varying results. In this manuscript, we report our experience with fludarabine (120 mg/m 2 ), very low dose cyclophosphamide (1200 mg/m 2 ) and antithymocyte globulin (7.5 mg/kg). Low dose total body irradiation (2 Gy) was added to the conditioning regimen for patients older than 15 years. Nineteen patients (median age 23 years) underwent transplant between 2008 and 2015. The majority (89%) were younger than 40 years. Stem cell source was BM ( n  = 11) or PBSC ( n  = 8). GvHD prophylaxis consisted of cyclosporine and either a short course of methotrexate ( n  = 9) or mycophenolate mofetil ( n  = 10). Eighteen (94.7%) patients achieved sustained engraftment. The median times to neutrophil and platelet engraftments were 19 (range: 14–34) and 17.1 (range: 12–25) days, respectively. The day-30 cumulative incidence of neutrophil and platelet engraftment was 89.4% and 94.7%, respectively. No secondary graft rejection was observed. The 1-year cumulative incidence of aGvHD (grade II–IV) and cGvHD was 11.7% and 0%, respectively. The 2-year GvHD-free survival rate was 78.6% (95% CI: 52.5–91.4%). Fludarabine-based reduced intensity regimen for MRD allo-HSCT in SAA compares favorably to other available regimens. This regimen deserves further investigations with larger cohort of patients.