脐血外周血造血干细胞移植治疗遗传性溶血性贫血的疗效比较

Analysis of clinical outcome between allogeneic peripheral blood and umbilical cord blood stem cell transplantation for hereditary haemolytic anemia

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 outcome in patients with hereditary haemolytic anemia (HHA) between allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) and umbilical cord blood stem cell transplantation (UCBT).Methods Sixteen patients with HHA (14 β-thalassemia major,2 congenital idiopathic haemolytic anemia) received PBSCT (n=7) or UCBT (n=9),respectively.The conditioning regimen consisted of busulfan 14～20mg/kg,cyclophosphages 160～200mg/kg and antithymocyte globulin 90 mg/kg for all patients.Of them,8 patients also received melphalan (90mg/m 2),2 had thiotepa (6mg/kg) and another 4 thiotepa with fludarabine ( 150mg/m 2).Results The median follow-up period was 49(38～64 ) months in UCBT group and 39(25～49) months in PBSCT group.Seven patients with UCBT and 6 with PBSCT received engraft successfully,of them 2 relapsed within 60 days post UCBT,but none relapsed in the PBSCT group.Transplant-related death occurred in 1 patient in UCBT group,and 2 in PBSCT group,respectively.The cause of death was acute renal failure in UCBT,while in PBSCT,one of them died of hepatic veno-occlusive disease and the other got Escherichia coli septicemia.The aGVHD occurred in 4 patients in UCBT group and 5 in the PBSCT group,while cGVHD did in 1 and 3,respectively.Conclusion The high engraftment rate of PBSCT is its advantage,and the high risk of GVHD is its disadvantage.The lower rate and mildness of GVHD is the advantage of UCBT.Furthermore,the transplantation with HLA mismatched can be used successfully.However,the lower engraftment is the disadvantage of UCBT.