人类白细胞抗原单倍型相合外周血造血干细胞移植治疗血液系统疾病的疗效与安全性

The efficacy and safety of unmanipulated haploidentical peripheral blood stem cell transplantation on hematologic diseases

目的 评价非去T细胞人类白细胞抗原（HLA）单倍型相合外周血造血干细胞移植治疗血液系统疾病的疗效与安全性.方法 回顾性分析2007年7月-2011年12月在解放军总医院行HLA单倍型相合外周血造血干细胞移植的病例资料.结果 49例移植受者中位年龄22（3 ～59）岁.供受者HLA配型5/10相合15例,6/10相合11例,7/10相合7例,8/10相合5例,9/10相合11例.输注的单个核细胞中位数为10.01 （7.05 ～ 25.34）×108/kg,CD34＋细胞中位数为4.51 （2.01 ～11.47）×106/kg.1例复发难治型白血病患者植入失败.髓系造血重建中位时间为14（10～25）d,血小板重建中位时间为22（10～135）d.移植后中位随访时间347（7～ 1765）d.移植后100 d急性移植物抗宿主病（aGVHD）累计发生率为（61.6±7.3）％,Ⅱ～Ⅳ度aGVHD累计发生率为（28.6±6.7）％.2年慢性移植物抗宿主病（cGVHD）累计发生率为（42.6±8.5）％,广泛性cGVHD累计发生率为（22.7±7.6）％.100 d移植相关死亡累计发生率为（14.7±5.1）％,2年移植相关死亡累计发生率为（30.9±8.8）％；移植后2年累计复发率为（25.4±7.0）％.2年累计总生存率和无病生存率分别为（58.1±8.8）％和（53.9±8.4）％.结论 非去T细胞亲缘HLA单倍型相合外周血造血干细胞移植是一种有效的造血干细胞移植方式.

Objective To evaluate the efficacy and safety of unmanipulated haploidentical allogeneie peripheral blood stem cells transplantation （PBSCT） on hematologic diseases. Methods Patients who underwent unmanipulated HLA-mismatched/haploidentical PBSCT from July 2007 to December 2011 were investigated retrospectively. Results Forty-nine patients with hematologic diseases underwent unmanipulated human leukocyte antigen （HLA）-mismatched/haploidentieal PBSCT with myeloablative conditioning. All patients were mismatched at the allele level for HLA-A, HLA-B, HLA-Cw, HLA-DRB1 and HLA-DQ1. Fifteen patients were mismatched in 5 loci, 11 patients in 4 loci, 7 patients in 3 loci, 5 patients in 2 loci, and 11 patients in 1 locus. The median numbers of mononuelear cells and CD34 cells infused at transplantation were 10. 01 （7.05-25.34） x 10S/kg and 4. 51 （ 2. 01-11.47 ） x 106/kg, respectively. Patients achieved myeloid and platelet engraftment at a median of 14 （10-25） days and 22（10-135） days, respectively. The cumulative incidence of acute graft versus host disease （aGVHD） on day 100 was （ 61.6 ± 7.3 ） %, and the 2-year cumulative incidence of chronic graft versus host disease （cGVHD） was （42. 6 ± 8. 5） %. One hundred-day transplantation related mortality （TRM） rate and 2-year cumulative TRM rate were （ 14. 7 ± 5. 1 ） % and （ 30. 9 - 8. 8 ） %, respectively. The 2-year cumulative incidence estimate of relapse was （25.4 ± 7. 0） %. The 2-year cumulative overall survival rate was （58. 1 ± 8.8 ） % and 2-year disease-free survival rate was （ 53.9 ± 8.4 ） % with an 11.5-months median follow-up. Conclusion Unmanipulated PBSCT is a promising protocol for patients with hematologic diseases in HLA- mismatched/haploidentieal transplant settings.