HLA半相合异基因外周血造血干细胞移植治疗AA克隆演变继发MDS疗效分析

Efficiency and safty of haploidentical hematopoietic stem cell transplantation for myelodysplastic syndrome evolved from aplastic anemia

目的:回顾性分析应用HLA半相合异基因外周血造血干细胞移植(haploidentical hematopoietic stem cell transplantation,haplo-HSCT)治疗再生障碍性贫血(aplastic anemia,AA)克隆演变继发骨髓增生异常综合征(secondary myelodysplastic syndrome,s MDS)的疗效和安全性。方法:本中心5例AA患者经强化免疫抑制治疗后演变为s MDS,均采用haplo-HSCT,观察植入情况,移植物抗宿主病(graft versus host disease,GVHD)、移植相关并发症和移植相关病死率(transplant related modify,TRM)、总体生存(overall survival,OS)时间等。结果:5例中位OS时间63个月(41.9~149.3个月),移植后中位OS时间12.9个月(2.4~36.5个月),1年累积生存率60%,TRM 40%。移植后粒系植入时间18 d(14~22 d),血小板植入时间21 d(15~65 d),总植入率100%。60%(3/5)患者发生Ⅰ~Ⅲ度急性移植物抗宿主病(acute graft versus host disease,a GVHD),无Ⅳ度a GVHD发生,20%(1/5)患者发生慢性移植物抗宿主病(chronic graft versus host disease,c GVHD)。中位随访时间63个月(41.9~149.3个月)。结论:在无合适HLA相合供者情况下,haplo-HSCT可作为AA演变MDS患者的有效治疗策略。

Objective：To evaluate the efficiency and safety of haploidentical hematopoietic stem cell transplantation （haplo-HSCT） for treatment of secondary myelodysplastic syndrome （sMDS） after AA. Methods： Evaluation of therapeutic effect, incidence rate of aGVHD and cGVHD, complications and overall survival （OS） of 5 AA post MDS patients who received haplo-HSCT in this center, and compared with other treatments for sMDS evolved from AA. Results：Among 5 patients, median OS was 63 months （range from 41.9 to 149.3 months）, OS after HSCT was 12.9 months （range from 2.4 to 36.5 months）. 1-year event free survival was 60%. All patients achieved neutrophil and platelet engraftment at a median of 18 days（range from 14 to 22 days）and 21 days（range from 15 to 65 days） respectively. 3 occurred Ⅰ-Ⅲ° acute graft versus host disease （aGVHD）, and 1 with chronic graft versus host disease （cGVHD）. Medial follow-up was 63 months（range from 41.9 to 149.3 months）. Conclusion：The haplo-HSCT can be an opition for sMDS from AA patients in case of an HLA-identical donor is not available.