摘要
目的探讨非冻存同胞脐血干细胞移植治疗儿童重型地中海贫血的安全性和优势。方法回顾2016年10月至2021年6月在厦门大学附属中山医院因重型β地中海贫血接受非冻存同胞全相合脐血干细胞移植9例儿童受者的临床资料,分析总结其预处理方案、干细胞植入的过程及随访情况。结果9例儿童受者中男性5例,女性4例,年龄为4岁(2~11)岁,铁蛋白中位水平为2997μg/L(1936~5512μg/L),9例儿童受者母亲在孕12~16周时行绒毛检测明确胎儿供者与儿童受者HLA配型全相合。接受氟达拉滨+白消安+环磷酰胺的预处理方案,移植后给予环孢素A+吗替麦考酚酯+/-短程甲氨蝶呤预防移植物抗宿主病(GVHD)。8例成功植入,1例原发性植入失败,中性粒细胞植入时间为19.5 d(15~26)d,血小板植入时间为32 d(22~34)d,红细胞植入时间为30.5 d(18~37)d。截止2021年9月1日,随访时间27个月(3~59个月),干细胞植入率为88.89%,无移植相关的死亡,总存活率100%,无地中海贫血存活率88.89%。2例发生Ⅱ级的皮肤急性GVHD(22.2%),无Ⅲ度以上急性GVHD发生,未出现慢性GVHD病例。发生EB病毒感染1例,未发生巨细胞病毒感染事件。结论非冻存同胞脐血干细胞移植治疗儿童地中海贫血具有干细胞移植植入成功率高、GVHD发生率低的优势。
Objective To explore the safety and advantages of non-cryopreserved sibling umbilical cord blood hematopoietic stem cell transplantation for major thalassaemia in children.Methods From October 2016 to June 2021,9 patients with major beta thalassaemia received non-cryopreserved hematopoietic stem cell transplantation of sibling umbilical cord blood at Zhongshan Hospital of Xiamen University.The pretreatment scheme,the process of stem cell implantation and follow-up were analyzed and summarized.Results Among the 9 cases,there were 5 males and 4 females with a median age of 4(2~11)years.Median level of ferritin was 2997(1936~5512)μg/L.At gestational weeks 12~16,each patient's mother underwent villi testing to confirm that the donor without thalassaemia major was complete HLA-matched with the patient.All of them received an intensive conditioning regimen made up of cyclophosphamide(CTX),fludarabine and busulfan(Bu).Graft-versus-host disease(GVHD)was prevented by cyclosporine A(CSA)and mycophenolate mofetil(MMF)with or without methotrexate(MTX).Except for one failed implant,8 cases were successfully engrafted.Median time of neutrophil implantation was 19.5(15~26)days,median time of platelet implantation 32(22~34)days and median time of erythrocyte implantation 30.5(18~37)days.Up until September 1,2021,the median follow-up period was 27(3~59)months and the rate of successful engraftment 88.89%.There was no transplant-related mortality.Overall survival was 100%and thalassaemia-free survival 88.89%.Two patients developed gradesⅡskin acute GVHD(22.2%).No gradeⅢ-ⅣGVHD or chronic GVHD occurred.Epstein-Barr virus infection occurred in 1 case.No infection of cytomegalovirus occurred.Conclusions For major thalassaemia in children,stem cell transplantation of non-cryopreserved sibling cord blood is both safe and feasible with a high implantation rate and a low incidence of GVHD.
作者
陆婧媛
林进宗
陈凌
洪秀理
陈亚玫
石洁
鹿全意
Lu Jingyuan;Lin Jinzong;Chen Ling;Hong Xiuli;Chen Yamei;Shi Jie;Lu Quanyi(Department of Hematology,Xiamen university Zhongshan hospital,Xiamen 361004,China)
出处
《中华器官移植杂志》
CAS
2022年第3期151-155,共5页
Chinese Journal of Organ Transplantation
基金
福建省自然基金引导性项目(2019D009)。
关键词
造血干细胞移植
地中海贫血症
儿童
Hematopoietic stem cell transplantation
Thalassemia
Child
