Objective: To study the outcome of HLA-identical hematopoietic stem cell transplantation (HSCT) for severe aplastic anemia (SAA).
Methods: Twenty patients diagnosed with SAA received allogeneic HSCT from HLA-identical donors (17 from siblings and 3 from unrelated donors) between January 2000 and November 2008. Conditioning regimen consisted of cyclophosphamide (Cy) and anti-thymocyte immunoglobulin (ATG). The patients were administrated with G-CSF-primed bone marrow(G-BM) and mobilized peripheral blood (G-PB) as grafts from the sibling donors or only G-PB from the unrelated donors.
Results: The median infused number of mononuclear cells and CD(34)(+) cells were 7.89 (4 - 14.21) x 10(8)/kg and 2.60 (0.81 - 4.45) x 10(6)/kg. All the patients got engraftment with 100% donor chimerism. The median time of neutrophil and platelet engraftment were 14 (11 - 20) d and 12 (8 - 108) d respectively. The cumulative incidence rate of acute GVHD at 100 d was 16% (grade I: 3 cases, grade II: 3 cases). Chronic GVHD occurred in 7 of the 19 evaluable cases (4 limited, 3 extensive). Till February 28, 2009, with a median follow-up of 18 months, 17 patients were alive and the overall survival rate was 82.5%.
Conclusion: The study confirms that using G-PB with or without G-BM as graft after Cy+ATG conditioning results in excellent outcome of HLA-identical HSCT in patients with SAA.