非亲缘关系脐带血移植治疗25例血液系统疾病患者的临床研究  

Clinical Study of Unrelated Umbilical Cord Blood Transplantation for 25 Patients with Hematological Diseases

作　　者：常惠荣[1] 程珍[1] 陈广华[1] 孙爱宁[1] 乔曼[1] 马骁[1] 王荧[1] 陈峰[1] 金焕英 陆瑶 张嘉卿 付琤琤[1] 金正明[1] 唐晓文[1] 韩悦[1] 仇慧英[1] 吴德沛[1] 

机构地区：[1]苏州大学附属第一医院,江苏省血液研究所,卫生部血栓与止血重点实验室,江苏苏州215006 [2]上海市脐带血造血干细胞库,上海200051

出　　处：《中国实验血液学杂志》2014年第3期767-773,共7页Journal of Experimental Hematology

基　　金：江苏高校优势学科建设工程资助项目(2010);江苏省临床医学中心项目(ZX201102);国家自然科学基金项目(81270617);上海市科技创业中心创新基金项目(1002H103000)

摘　　要：本研究观察非亲缘脐带血造血干细胞移植(UCBT)在血液系统疾病患者的植入率、移植物抗宿主病(GVHD)、移植相关死亡(TRM)、复发和生存情况。25例接受UCBT的血液系统疾病患者(儿童8例、成人17例)中。3例接受单份脐带血(CB),22例接受双份脐带血。供/受体低分辨人类白细胞抗原(HLA)配型结果为9例6/6位点相合、16例4-5/6位点相合。在预处理方案中21例采用TBI/Cy±Flu±ATG或BuCy±Flu±ATG,4例采用FC+ATG方案。移植物抗宿主病(GVHD)的预防采用以环孢素(CsA)为主的方案。结果表明,存活超过42 d的20例患者中16例(80.0%)获得植入;粒系植入至移植后42 d的累积发生率为64.0%,中位时间17.0 d;巨核系植入至移植后100 d的累积发生率为60.0%,中位时间35.0 d;II-IV度、III-IV度急性GVHD(aGVHD)至移植后100 d的累积发生率分别为44.0%和30.7%;截止至随访结束日期,移植相关死亡率(TRM)的累积发生率为54.3%;25例患者的总生存率为42.7%;17例可评估恶性血液病患者中7例(41.2%)存活,且仅有1例复发,无事件生存率(EFS)为35.3%;5例可评估的非恶性血液病患者中4例存活,2例处于持续脐血植入状态,2例自身造血恢复;处于疾病进展期的3例患者中仅1例患者存活。结论:低分辨HLA-4-6/6位点相合的UCBT治疗血液系统疾病是可行的,双份脐血移植(dUCBT)可以克服单份脐血细胞数量不足的缺点,可用于治疗大体重儿童及成人患者。This study was purposed to investigate the engraftment, graft-versus-host disease （GVHD）, transplantation related mortality （TRM）, relapse and survival in hematologic patients received unrelated umbilical cord blood transplantation （UCBT）. A total of 25 patients with hematological disease underwent UCBT, including 8 pediatric and 17 young adult patients. Among them 3 cases received single unit of UCBT and 22 cases received double units of UCBT. For donor/recipients human leukocyte antigen （HLA） matching： HLA 6/6 loci matched in 9 cases, HLA 4 -5/6 loci matched in 16 cases. There were 19 patients with hematologic malignancies, including 3 cases in the period of disease progression and 6 cases of non-hematologic malignancies. Conditioning regimens were TBI/Cy ＋ Flu ＋ ATG or BuCy ＋ Flu ＋ ATG for 21 patients and Cy ＋ Flu ＋ ATG for 4 patients. For prophylaxis of acute graft-versus-host disease （aGVHD） the regimen of cyclosporine （CsA） as dominant drug was used. The results showed that among 16 patients （ 80.0% ） achieved engraftment, 20 patients survived for more than 42 d after transplantation. The cumulative neutrophil recovery rate on day 42 after transplant was 64.0%, with a median time of 17.0 d;the cumulative platelet recovery rateon day 100 after transplant was 60.0 % ,with a median time of 35.0 d. The cumulative rate of grade Ⅱ - IV and Ⅲ - IV aGVHD after transplantation 100 d was 44.0% and 30. 7%, respectively. Until the end of the follow-up, the cumulative rate of TRM was 54. 3%. For all the patients, overall survival rate was 42. 7%. Out of 17 evaluable patients with hematologic malignancies 7 cases （41.2%） survived to date, and only 1 case relapsed, so event-free survival rate was 35. 3 %. Out of 5 evaluable patients with non-hematologic malignancies, 4 patients survived and 2 patients were in stable engraftment state, 2 cases with autologous hematopoietic recovery. Among 3 cases of hematologic malignancies at advanced stage, only 1 case survived to date. I

关 键 词：异基因造血干细胞移植 脐带血移植 血液系统疾病 

分 类 号：R457.7[医药卫生—治疗学]