Abstract

Objective To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for severe aplastic anemia (SAA).Method The clinical data of 70 cases of SAA receiving allo-HSCT from January 2003 to February 2012 were retrospectively analyzed.There ere 40 males and 30 females with median age of 20 (5-41) years old.Fifty-one patients received HLA-matched sibling donor (MSD) allo-HSCT,and 19 patients received alternative donor(AD) alloHSCT.Of 51 patients receiving MSD allo-HSCT,20 patients were subjected to bone marrow transplantation (BMT),and 31 patients to peripheral blood stem cell transplantation (PBSCT).The median reinfusion quantity of CD34+ was 3.46 (2.00～4.80) × 106/kg in BMT,and 3.22 (1.27～ 5.98) × 106/kg in PBSCT.Of patients receiving AD allo-HSCT,10 patients were subjected to BMT,and 9 to PBSCT.The median reinfusion quantity of CD34+ was 3.10 (2.11 ～4.38) × 106/kg in BMT,and 4.90 (2.08～6.88) × 106/kg in PBSCT.Result Hematopoiesis reconstitution was achieved in all 70 patients,median follow up time was 35.5 (1 ～ 119) months,and prospective overall survival (OS) for 5 years was (79.0 ± 5.1) ％.Of the patients receiving MSD allo-HSCT,acute graftversus-host disease (aGVHD) occurred 16 cases,chronic GVHD (cGVHD) in 10 cases,and graft rejection (GR) in 6 cases.There were 7 deaths.OS for 5 years was (85.0 ± 5.4) ％.In the patients receiving AD allo-HSCT,aGVHD occurred in 12 cases,cGVHD in 7 cases,and GR in one case.There were 7 deaths.OS for 5 years was (63.2 ± 11.1)％.Multiplicity analysis revealed that PBSCT (P=0.049),severe (Ⅱ-Ⅳ) aGVHD (P =0.025) and severe infection (invasive fungal disease or cytomegalovirus) (P =0.05) could reduce the efficacy of allo-HSCT for SAA significantly.Conclusion Allo-HSCT is an effective therapy for patients with SAA,especially those receiving MSD allo-HSCT.AD allo-HSCT might be feasible for SAA patients without HLA-matched sibiling donors.Bone marrow transplantation which can effectively prevent and treat severe (Ⅱ-Ⅳ) aGVHD and severe infection (invasive fungal disease or cytomegalovirus) after transplantation may improve the efficacy of allo-HSCT for SAA. Key words: Anemia aplastic, severe; Stem cell transplantation; Hematopoietic ; Donors ; Sibling

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