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目的 比较亲属异基因脐血造血干细胞移植 (UCBT)与亲属异基因外周血造血干细胞移植 (PBSCT)治疗遗传性溶血性贫血 (溶贫 )的临床疗效。方法 16例遗传性溶贫 (重型 β地中海贫血 14例 ,先天性特发性溶血性贫血 2例 )中 9例行UCBT ,7例行PBSCT。预处理方案以白消安 14～ 2 0mg/kg、环磷酰胺 16 0～ 2 0 0mg/kg和抗胸腺细胞球蛋白 90mg/kg为基础 ,8例加马法兰 90mg/m2 ,2例加噻替哌 6mg/kg ,4例加噻替哌和氟达拉宾15 0mg/m2 。结果 UCBT组植入 7例 (7/ 9) ,其中 2例于移植术后 6 0d(+6 0d)内排斥、回复地中海贫血 (地贫 )状态 ,1例发生急性肾衰竭死亡 ;4例出现急性移植物抗宿主病 (aGVHD) ,1例出现慢性移植物抗宿主病 (cGVHD) ;中位随访时间 4 9个月 (38～ 6 4个月 )。PBSCT组植入 6例 (6 / 7) ,无继发排斥 ,1例发生肝衰竭死亡 ,1例于 +3d死于败血症 ;5例出现aGVHD ,其中 3例延续为cGVHD ;中位随访时间 39个月 (2 5～ 4 9个月 )。结论 UCBT、PBSCT治疗遗传性溶贫具有不同的特点。PBSCT治疗具有植入率高 ,GVHD发生率也高的特点。UCBT具有GVHD程度较轻 ,提示人类白细胞抗原配型不全相合也可开展移植 ,但应改善低植入率的问题。
Objective To compare the clinical efficacy of allogeneic cord blood hematopoietic stem cell transplantation (UCBT) and relative allogeneic peripheral blood stem cell transplantation (PBSCT) in the treatment of hereditary hemolytic anemia (PFS). Methods Nineteen patients with hereditary lymphadenosis (14 with severe β-thalassemia and 2 with congenital idiopathic hemolytic anemia) underwent UCBT in 9 and PBSCT in 7. The pretreatment regimen was based on busulfan 14 ~ 20mg / kg, cyclophosphamide 160 ~ 200mg / kg and anti-thymocyte globulin 90mg / kg, 8 cases of melphalan 90mg / m2, Piperazine 6mg / kg, 4 cases plus thiotepa and fludarabine 150mg / m2. Results In the UCBT group, 7 cases (7/9) were implanted, of which 2 cases were rejected within 60 days (+ 60 days) after transplantation, and thalassemia (thalassemia) was restored and 1 case died of acute renal failure. Acute graft versus host disease (aGVHD) and chronic graft versus host disease (cGVHD) occurred in 1 case; median follow-up was 49 months (ranged from 38 to 64 months). One patient died of liver failure and one died of sepsis at + 3d. Five patients had aGVHD, of which 3 patients continued as cGVHD. The median follow-up time was 39 Month (25 ~ 49 months). Conclusions UCBT and PBSCT have different characteristics in the treatment of hereditary lean mass. PBSCT treatment has the characteristics of high implantation rate and high incidence of GVHD. UCBT has a low degree of GVHD, suggesting that human leukocyte antigen incompatibility can also be transplanted, but should improve the low implantation rate.