Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders

Allogeneic hematopoietic stem cell transplantation (allo-hsct) from a geno-identical matched sibling (msd) is one of the most successful therapies in patients with non-malignant hematological disorders. this study included 273 patients with severe aplastic anemia (saa),152 patients with b-thalassemia major (btm),31 patients with fanconi's anemia (fa),20 patients with congenital immunodeficiency diseases (id),and 13 patients with inherited metabolic disorders (imd) allografted from a msd. in saa,the 8-year overall survival (os) of the whole group patients was 74%. os was significantly better in patients conditioned with fludarabine and cyclophosphamide (flu/cy) than in those who received cyclophosphamide and antithymocyte globulin (cy/atg) (. p=. 0.021). acute graft-versus-host disease (agvhd) grade ii-iv occurred in 15% while chronic gvhd (cgvhd) occurred in 28%. in btm,the 12-year disease-free survival (dfs) of the whole group of btm patients was 72.4%. dfs was 74% for peripheral blood stem cell (pbsc) group compared to 64% in the bm stem cell group. the incidence of graft rejection was significantly lower in patients who received pbsc than in those who received bm (9% vs 25%) (. p=. 0.036). agvhd grade ii-iv and cgvhd occurred in 15% and 12% of the whole group of btm patients respectively. in fa,the 5-year os was 64.5%. graft rejection occurred in 10% of patients. grade ii-iv agvhd occurred in 16% while cgvhd occurred in 4%. in id,the 5-year os was 62%. graft rejection occurred in two (10%) patients. three patients (15%) developed grade ii-iv agvhd,2 of them progressed to secondary cgvhd. in imd,os was 46% at 5. years. graft rejection occurred in 8% of patients. agvhd grade ii-iv occurred in 15% while cgvhd occurred in 14%. in conclusion,allo-hsct provides a higher dfs rate over conventional therapies for patients with non-malignant hematological disorders with prolonged survival. © 2014.