亲缘单倍体非体外去除T细胞的造血干细胞移植和同胞全相合移植治疗儿童急性白血病的临床研究

目的：探讨单倍体非体外去除T细胞的造血干细胞移植治疗儿童急性白血病的临床疗效。 方法：回顾性分析2007年6月8日至2015年5月4日于首都医科大学附属儿童医院采用亲缘单倍体非体外去除T细胞的造血干细胞移植治疗儿童急性白血病23例（亲缘单倍体组），同胞全相合移植21例（同胞全相合组）。两组的预处理方案均为清髓性，回输集落刺激因子动员的造血干细胞，采用环孢素、甲氨蝶呤±霉酚酸酯联合方案预防移植物抗宿主病（GVHD），采用低分子肝素±前列腺素E预防静脉闭塞病。 结果：两组患儿均获得完全、持久的供者造血干细胞植入。亲缘单倍体组和同胞全相合组：①WBC植入中位时间分别为（13.1±2.3）和（12.5±1.4）d，PLT植入的中位时间分别为（19.4±8.0）和（22.9±7.7）d，差异均无统计学意义（P>0.05）；②中位随访时间分别为(49.6±30.3)和(32.5±31.6)月，移植后总体生存率分别为69.5%（16/23）vs 71.4%（15/21），差异无统计学意义（P>0.05）；③亲缘单倍体相合组与同胞全相合组急性GVHD临床分级（Ⅰ~Ⅳ）65.2%（15/23）和23.8%（5/21），差异有统计学意义（P<0.05）。 结论：亲缘单倍体非体外去除T细胞与同胞全相合造血干细胞移植治疗儿童急性白血病的安全性与疗效相似。

A clinical study of haploidentical and matched sibling donor hematopoietic stem cell transplantation without in vitro T-cell depletion for the treatment of acute leukemia in pediatric patients

Objective：To investigate the clinical outcome of haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion for children with acute leukemia. Methods：In Beijing Children's Hospital, Capital Medical University, from June 8, 2007 to May 4, 2015, 23 children with acute leukemia had been treated with haploidentical related hematopoietic stem cell transplantation without in vitro T-cell depletion, and 21 children with acute leukemia had been treated with matched sibling donor hematopoietic stem cell transplantation. Both groups used myeloablative preconditioning regime, and G-CSF mobilized hematopoietic stem cell. Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine A, methotrexate with or without mycophenolate mofetil. To prevent hepatic vein occlusion disease (VOD), prostaglandin E combined with or without low molecular weight heparin was used. Results：All patients attained hematopoietic stem cell transplantation successfully. The median time of leukocyte recovery for haploidentical group and matched sibling group were (13.1 ±2.3) days and (12.5 ± 1.4) days, respectively(P>0.05). The median time of platelet recovery for haploidentical group and matched sibling group were (19.4 ± 8.0) days and (22.8 ± 7.7) days, respectively (P>0.05). The median follow-up time was (49.6 ± 30.3) months and (32.5 ± 31.6) months after transplantation, the overall survival rates were 69.5% (16/23) vs71.4% (15/21), the difference was not statistically significant (P>0.05). Acute GVHD clinical grading (Ⅰ~Ⅳ) was 65.2% (15/23) in haploidentical group and 23.8% (5/21) in matched sibling group, and the difference was statistically significant (P<0.05). Conclusion：It can be achieved the same clinical effect of haploidentical related hematopoietic stem cell transplantation without in vitro T-cell depletion in the treatment of children with leukemia, compared with matched sibling hematopoietic stem cell transplantation. The safety and efficacies of haploidentical group were close to the matched sibling group.