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目的:探讨重症联合免疫缺陷(SCID)移植的经验。方法:对我院4例X-连锁SCID患者进行了5次造血干细胞移植(HSCT)。供者采用人类白细胞抗原(HLA)全相合同胞骨髓1例次,母亲半相合CD34+外周造血干细胞2例次,无关脐血2例次。2例次脐血移植者应用了清髓性预处理,并进行移植物抗宿主病(GVHD)预防,其余3例次未进行预处理和GVHD预防。结果:所有患者在活动性感染的基础上接受HSCT。骨髓和外周血移植物平均CD34+细胞为6.45×107/kg,脐血为1.38×106/kg。3例患者移植后2周左右出现Ⅰ～Ⅱ度急性GVHD。最终2例因移植后肺部感染加重死亡,1例因黄疸、肝功能不良放弃治疗,仅1例接受再次脐血移植者经历了首次移植失败、重度肝静脉阻塞病(VOD)、重症肺炎后幸存,该患儿移植后78d自然杀伤(NK)细胞数量接近正常,10个月免疫球蛋白数量恢复正常,13个月T细胞数量接近正常,目前已经随访到移植后15个月。结论:HSCT治疗SCID具有可行性,早期诊断并在严重感染前进行移植是提高移植成功的关键。
Objective: To explore the experience of severe combined immunodeficiency (SCID) transplantation. Methods: Four hematopoietic stem cell transplants (HSCT) were performed in 4 X-linked SCID patients in our hospital. The donors used human leukocyte antigen (HLA) as a complete complement with sarcopenic bone marrow in 1 case, maternal half matched CD34 + peripheral blood stem cell in 2 cases, unrelated cord blood in 2 cases. Two cases of umbilical cord blood transplants used myeloablative pretreatment, and graft-versus-host disease (GVHD) prevention, the remaining three cases without pretreatment and prevention of GVHD. Results: All patients received HSCT on the basis of active infection. The mean CD34 + cells in bone marrow and peripheral blood grafts were 6.45 × 107 / kg and cord blood was 1.38 × 106 / kg. Three patients had grade Ⅰ ~ Ⅱ acute GVHD about 2 weeks after transplantation. The final two cases died of pulmonary infection after transplantation. One case was given up because of jaundice and poor liver function. Only one case of transplantable umbilical cord blood transplanted underwent the first graft failure, severe hepatic vein occlusion (VOD) and severe pneumonia Survival, the number of natural killer (NK) cells in the children 78 days after transplantation close to normal, 10 months the number of immunoglobulin returned to normal, 13 months the number of T cells close to normal, has been followed up 15 months after transplantation. CONCLUSION: HSCT is feasible for the treatment of SCID. Early diagnosis and transplantation before severe infection are the keys to successful transplantation.