论文部分内容阅读
目的:评估异基因造血干细胞移植(allo-HSCT)治疗重型再生障碍性贫血(SAA)Ⅱ型的疗效和安全性。方法:回顾性分析接受allo-HSCT治疗的22例SAAⅡ患者的临床资料,其中同胞相合供者移植10例,无关供者移植12例;18例行外周血造血干细胞移植,4例行骨髓联合外周血造血干细胞移植。预处理方案为环磷酰胺+抗人胸腺细胞球蛋白±氟达拉滨±白消安±低剂量TBI;采用环孢霉素A或他克莫司、短疗程甲氨蝶呤±霉酚酸酯预防移植物抗宿主病(GVHD)。回输单个核细胞中位数13.55(5.12~25.90)×10~8/kg,CD34~+细胞中位数7.30(2.19~40.32)×10~6/kg。结果:20例(90.91%)患者获得造血重建,可评估患者的中性粒细胞和血小板的中位植入时间分别为12(9~22)d和13(9~28)d。移植后2年急性GVHD、慢性GVHD和移植排斥、移植相关死亡累积发生率分别为40.00%、30.00%、9.09%和22.73%。细菌血流感染率22.73%,肺部侵袭性真菌病发生率40.91%,巨细胞病毒和EBV感染率分别为75.00%和50.00%;心、肝、肾功能不全发生率分别为45.45%、13.64%和36.36%。中位随访23(10~68)个月,17例患者生存,预期2年总生存率77.27%,预期2年无病生存率72.73%。单因素分析结果显示,移植后发生严重(Ⅱ~Ⅳ度)急性GVHD和重要脏器功能不全可显著降低allo-HSCT治疗SAAⅡ的疗效(P=0.018、0.009)。结论:同胞相合供者和无关HLA匹配供者alloHSCT是治疗SAAⅡ的有效手段。
Objective: To evaluate the efficacy and safety of allo-HSCT in the treatment of type Ⅱ severe acute aplastic anemia (SAA). Methods: The clinical data of 22 patients with SAA Ⅱ who underwent allo-HSCT were retrospectively analyzed. Among them, 10 matched sibling donors and 12 unrelated donors; 18 received peripheral blood stem cell transplantation; 4 received combined bone marrow Blood stem cell transplantation. Pretreatment regimens were cyclophosphamide + anti-human thymocyte globulin ± fludarabine ± busulfan ± low-dose TBI; cyclosporin A or tacrolimus, short course methotrexate mycophenolic acid Ester prevents graft versus host disease (GVHD). The median of transfused mononuclear cells was 13.55 (5.12 ~ 25.90) × 10 ~ 8 / kg and the median of CD34 ~ + cells was 7.30 (2.19 ~ 40.32) × 10 ~ 6 / kg. RESULTS: Twenty patients (90.91%) had hematopoietic reconstructions. The median time to neutrophil and platelet countability was 12 (9-22) days and 13 (9-28) days, respectively. Two years after transplantation, the cumulative incidence of acute GVHD, chronic GVHD, and transplant rejection, graft-related deaths were 40.00%, 30.00%, 9.09% and 22.73%, respectively. The rate of bacterial bloodstream infection was 22.73%, lung invasive fungal disease was 40.91%, cytomegalovirus and EBV infection rates were 75.00% and 50.00% respectively. The rates of heart, liver and renal insufficiency were 45.45% and 13.64% And 36.36%. At a median follow-up of 23 months (range, 10-68 months), 17 patients survived, with a 2-year overall survival of 77.27% and a 2-year disease-free survival of 72.73%. Univariate analysis showed that severe (Ⅱ ~ Ⅳ) acute GVHD and dysfunction of vital organs after transplantation could significantly reduce the efficacy of allo-HSCT in the treatment of SAAⅡ (P = 0.018,0.009). CONCLUSION: AlloHSCT with sibling donor and unrelated HLA matched donor is an effective treatment for SAAⅡ.