非清髓异基因造血干细胞移植治疗骨髓增生异常综合征的疗效观察  

Transplantation of non-myeloablative allogeneic hematopoietic stem cells for myelodysplastic syndrome

作　　者：董征[1] 乔建辉[1] 余长林[1] 孙琪云[1] 胡锴勋[1] 艾辉胜[1] 孙雪东[1] 姚波[1] 郭梅[1] 

机构地区：[1]解放军307医院血液科,北京100071

出　　处：《解放军医学杂志》2014年第1期48-51,共4页Medical Journal of Chinese People's Liberation Army

基　　金：国家"863计划"生物和农业技术领域项目(2002AA216081)~~

摘　　要：目的探讨非清髓异基因造血干细胞移植(NST)治疗骨髓增生异常综合征(MDS)的疗效。方法 14例有血缘相关HLA相合的MDS患者接受NST治疗。骨髓增生异常综合征-难治性贫血(MDS-RA)患者非清髓预处理方案为:氟达拉滨(Flud)+环磷酰胺(CTX)+抗人胸腺细胞免疫球蛋白(ATG);骨髓异常综合征-难治性贫血伴原始细胞过多(MDSRAEB)患者非清髓预处理方案为:在MDS-RA患者非清髓预处理方案基础上加用马利兰(Bu)。移植物抗宿主病(GVHD)预防采用环孢素(CSA)联合霉酚酸酯(MMF)。结果 13例患者造血均获得快速重建,1例造血未恢复。中性粒细胞在术后第9～12天恢复到0.5×109/L以上,12例患者血小板在术后第11～21天恢复到30×109/L以上。14例中12例完全植入,2例混合嵌合植入,其中1例经过二次移植后达到完全植入,另1例在移植后2个月发生移植排斥,移植后70d因脑出血死亡。1例发生急性Ⅱ度肠道GVHD,经静脉给予他克莫司(FK506)和甲泼尼龙治疗后病情控制,但患者在后期应用FK506治疗过程中出现癫痫持续发作,经多种抗癫痫药物治疗未见好转,家属放弃治疗自动出院后死亡。14例中未见慢性GVHD和间质性肺炎发生。随访36～133个月,11例患者现无病存活,其中10例存活60个月以上,3例死亡。结论 NST治疗MDS安全可行,可为治愈MDS提供新手段。Objective To investigate the therapeutic effect of transplantation of non-myeloablative allogeneic hematopoietic stem cells in the treatment of myelodysplastic syndrome. Methods Fourteen patients with myelodysplastic syndrome received non-myeloablative allogeneic hematopoietic stem cells from HLA matched donors. The treatment regime consisted of cyclophosphamide, fludarabine, cyclosporine, anti-thymocyte globulin with or without busulfan. Graft-versus-host- disease （GVHD） prophylaxis was performed with cyclosporine combined with mycophenolate mofetil. Results All the treatments were generally well tolerated and all patients received engraftment of the donor cells successfully. Engraftment was observed in 85.7% （12/14） patients with complete donor cells, and in i patient （7.1%） mixed chimerism was changed into full donor chimerism, and graft failure was 7.1% （1/14）. Hematopoiesis （WBC recovered to more than 0.5 x 109/L during postoperative 9 day to 12 day and platelet recovered to more than 30 x 109/L during postoperative 11 day to 21 day） was recovered. The incidence of acute GVHD （grade ]I ） was 7.1% （1/14） and no chronic GVHD was found. Interstitial pneumonia （IP） was not found in these fourteen patients. Overall survival rate was 78.6% during the follow-up time from 36 months to 133 months. Conclusion Transplantation of non- myeloablative allogeneic hematopoietic stein cell is safe and effective for the treatment of myelodysplastic syndrome.

关 键 词：非清髓预处理 异基因造血干细胞移植 骨髓增生异常综合征 

分 类 号：R551.31[医药卫生—血液循环系统疾病]