Abstract

目的评估减低剂量预处理异基因造血干细胞移植(allo-HSCT)对范可尼贫血(FA)患儿的治疗效果。方法回顾性分析4例接受allo-HSCT FA患儿的临床资料。结果4例FA患儿中男1例、女3例,移植时中位年龄为7(5~10)岁,非亲缘10/10 HLA全相合外周血干细胞移植2例,同胞10/10 HLA全相合骨髓联合外周血干细胞移植、非亲缘10/10 HLA全相合脐血干细胞移植各1例。采用白消安联合低剂量环磷酰胺(20 mg/kg)为基础的预处理方案。4例FA患儿均获得造血重建,中性粒细胞植活中位时间为11(9~15)d,血小板植活中位时间为12(8~28)d。1例患儿发生Ⅰ度急性移植物抗宿主病,1例患儿发生出血性膀胱炎,4例患儿均未发生肝静脉闭塞病。结论减低剂量预处理allo-HSCT治疗FA患儿是安全、有效的。

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