间充质干细胞联合单倍体异基因造血干细胞移植治疗27例重型再生障碍性贫血临床疗效

Clinical effect of cotransplantation of haploidentical hematopoietic stem cells and umbilical cord mesenchymal stem cells for patients with severe aplastic anemia

目的 回顾性分析重型再生障碍性贫血（severe aplastic anemia,SAA）行脐带间充质干细胞（umbilical cord mesenchymal stem cells,UC-MSCs）联合单倍体异基因造血干细胞移植（haploidentical stem cells transplantatio,haplo-HSCT）治疗的临床疗效.方法 总结解放军总医院第一附属医院血液科2007年1月-2013年12月住院收治的27例SAA患者进行UC-MSCs联合haplo-HSCT治疗的资料,观察移植后细胞植入、移植物抗宿主病（graft-versus-host disease,GVHD）发生率及严重程度和2年总生存率（overall survival,OS）.结果 干细胞全部植入成功,无MSC回输不良事件发生.白细胞及血小板植入时间分别为12（8～ 21）d及14（10～26）d;Ⅱ～Ⅳ度和Ⅲ/Ⅳ度aGVHD发生率分别为40.7％和22.2％,cGVHD发生率为50.0％,广泛性仅为15.4％;2年OS为76.3％.结论 UC-MSCs联合haplo-HSCT对无全相合亲缘或无关供者的SAA患者疗效可行有效,可作为SAA患者挽救治疗选择.

Objective To analyse the clinical effect of cotransplantafion of haploidentical hematopoietic stem cells and umbilical cord mesenchymal stem cells （UC-MSCs） for patients with severe aplastic anemia （SAA）. Methods Clinical data about 27 patients with haploidentical stem cells transplantation （haplo-HSCT） in our center from January 2007 to December 2013 were summarized. The engraftment, graft-versus-host disease （GVHD）, overall survival （OS）, disease/progression-free survival （PFS） and relapse after 2 years were observed. Results All patients given MSCs showed sustained hematopoietic engraftment without any adverse UC-MSC infusion-related reaction. The median times of transplantation of neutrophil 〉 0.50×10^9/L and platelet 〉 20× 10^9/L engraftment were 12.0 days （8-21 days） and 14.0 days （10-26 days）, respectively. Grade Ⅱ to IV and Ⅲ/ Ⅳ aGVHD was observed in 11 of 27 patients （40.7%） and 6 of 27 cases （22.2%）, respectively. Chronic GVHD was observed in 13 of 26 patients （50.0%） and was extensive in 4 patients （15.4%）. The probability that patients would attain 2-years overall survival （OS） was 76.3%. Conclusion Cotransplantation of haploidentical hematopoietic stem cells and UC-MSCs has good treatment effect to patients with SAA without HLA-identical sibling donors or unrelated donors, which is a feasible salvage approach.