以强效免疫抑制为基础的预处理非亲缘异基因造血干细胞移植治疗重型再生障碍性贫血1例并文献复习  

Unrelated donor hematopoietic stem cell transplantation for severe aplastic anemia using fludarabine,antithymocyte globulin based conditioning containing low-dose cyclophosphamide:one case report

作　　者：周铭[1] 李庆山[1] 毛平[1] 王顺清[1] 

机构地区：[1]广州医学院附属第一人民医院血液科,广州510180

出　　处：《临床血液学杂志》2012年第5期583-586,共4页Journal of Clinical Hematology

基　　金：广东省科技计划项目(No:2011B031800053)

摘　　要：目的:探讨以氟达拉滨(Flu)加抗胸腺细胞球蛋白(ATG)强效免疫抑制联合极小剂量环磷酰胺(CTX)(1 200mg/m2)预处理方案在再障非亲缘移植中的可行性。方法:对1例诊断重型再生障碍性贫血(SAA),CsA治疗4年无效并反复输注红细胞、血小板的26岁男性患者进行非亲缘异基因造血干细胞移植治疗,高分辨HLA基因型10/10相合,供者为女性,血型相同,干细胞来源为G-CSF动员的外周造血干细胞,预处理方案以Flu加ATG强效免疫抑制为基础,联合极小剂量CTX:CTX 300mg/m2×4d,Flu 30mg/m2×4d,ATG3.75mg/kg×4d,输注单个核细胞(MNC)12.12×108/kg,CD34+细胞13.26×106/kg。术后GVHD预防联合应用环孢素A、短程氨甲喋呤、霉酚酸酯。结果:造血重建良好,术后11dANC>0.5×109/L,术后11dPLT>20×109/L,术后16dPLT>50×109/L。移植后输血量:RBC 3U,PLT 4U。术后21d、100d骨髓XX/XY染色体检测为完全供者型嵌合。术后29d出现巨细胞病毒血症,经更昔洛韦治疗3周转阴,无VOD,现随访6个月,出现局限型cGVHD并控制,血常规正常稳定,Kamofsky评分100分。结论:以Flu、ATG强效免疫抑制为基础的预处理进行非亲缘异基因造血干细胞移植治疗SAA安全有效,并发症少。Objective:To investigate efficacy of unrelated donor HSCT for SAA using Flu,ATG based conditioning containing low-dose CTX(1 200 mg/m2).Method:A 26-year-old patient with acquired SAA for 4 years showed no response to CsA and was transfusion-dependent.Lacking an HLA-identical sibling donor,the patient was treated with HSCT from an unrelated female donor 10/10 matched at the genetic level.The stem cell source was G-CSF mobilised peripheral blood stem cells(PBSC).Dose of stem cells infused:Mononuclear cells(MNCs)12.12×108/kg,CD34+ cells 13.26×106/kg.The conditioning regimen used more immunosuppressive regimens in combination with very low-dose CTX:CTX 300 mg/m2×4 d+Flu 30 mg/m2×4 d+ATG 3.75 mg/kg×4 d.Graft-versus-host disease(GVHD)prophylaxis was composed of CsA,short-course MTX and MMF.Result:The absolute neutrophil count(ANC)achieved 0.5×109/L at day +11 after transplantation,the platelet count>20×109/L at day +11,the platelet count>50×109/L at day +16,FISH method using XY chromosome as a marker signed the completely donor chimeric type at day +21 and day +100.CMV antigenemia was positive at day +29 and turn negative after treated by ganciclovir for 3 weeks.The patient has survived without GVHD for 6 months.Conclusion:Unrelated donor HSCT for SAA using Flu,ATG based conditioning containing low-dose CTX is safe and feasible,can be a choice for patients without a matched related donor.

关 键 词：贫血 再生障碍性 重型 造血干细胞移植 非清髓 非亲缘供者 

分 类 号：R556[医药卫生—血液循环系统疾病]