脐血间充质干细胞在难治性重型再生障碍性贫血异基因造血干细胞移植中的应用

The role of mesenchymal stem cells in allogeneic hematopoietic stem cell transplantation for patients with refractory severe aplastic anemia

目的 评价脐血间充质干细胞(UC-MSC)在难治性重型再生障碍性贫血(RSAA)异基因造血干细胞移植(allo-HSCT)中的疗效及安全性.方法 回顾性分析郑州大学附属肿瘤医院2010年3月至2018年7月间接受UC-MSC联合同胞相合供者及无关供者)外周血干细胞移植的25例RSAA患者,其中同胞相合供者组10例,无关供者组15例.结果 25例患者中男13例,女12例.1例患者原发性植入失败,1例患者血小板植入不良,其余23例患者成功植入.粒细胞、血小板植入的中位时间分别为12.5(10~23)d、15(11~25)d.Ⅰ/Ⅱ度急性GVHD、慢性GVHD发生率分别为37.5%(9/24)、21.7%(5/23),未发生重度GVHD和严重移植相关并发症.中位随访22.9(1.6~107.8)个月,25例患者中4例死亡,21例(84%)存活,移植后5年总生存率为(83.6±7.5)%.结论 UC-MSC联合allo-HSCT治疗RSAA疗效确切、安全性好.

Objective To evaluate the efficacy and safety of mesenchymal stem cells in allogeneic hematopoietic stem cell transplantation for patients with refractory severe aplastic anemia(R-SAA).Method The clinical data of 25 R-SAA patients receiving co-transplantation of mesenchymal stem cells combined with peripheral blood stem cells from sibling donors(10 cases)and unrelated donors(15 cases)from March 2010 to July 2018 in Zhengzhou University Affiliated Tumor Hospital were retrospectively analyzed.Antithymocyte globulin(ATG)treatment was ineffective/relapsed in 11 cases,and cyclosporine(CsA)treatment ineffective/relapsed in 14 cases.Results There were 13 male and 12 female among these patients.One patient had a primary graft failure,one patient had a poorly engraftment of platelets,and the remaining 23 patients achieved hematopoietic engraftment.The median time of granulocyte engraftment was 12.5(10-23)days and 15(11-25)days for megakaryocyte.Incidences of gradeⅠ/Ⅱacute graft-versus-host disease(aGVHD)and chronic graft-versus-host disease(cGVHD)were 37.5%(9/24)and 21.7%(5/23),respectively.There was no severe GVHD and no severe complications that related to transplantation.21 of 25(84%)patients were alive with a median follow-up of 22.9(1.6-107.8)months.The 5-year overall survival rate after transplantation was(83.6±7.5)%.Conclusion The combination of mesenchymal stem cells is reliable and safe in the treatment of R-SAA in peripheral blood stem cell transplantation of unrelated donors and sibling donors,which could significantly reduce the incidence of GVHD and severe transplantation-related complications.