免疫治疗桥接二次非血缘外周血干细胞移植治疗重型β地中海贫血一例  

Immunotherapy bridging secondary unrelated peripheral blood stem cell transplantation for severe thalassaemia:a case report and literature review

作　　者：阙丽萍[1] 徐宏贵[1] 詹丽萍[1] 李欣瑜[1] 方建培[1] 黄绍良[1] 黄科[1] Que Liping;Xu Honggui;Zhan Liping;Li Xinyu;Fang Jianpei;Huang Shaoliang;Huang Ke(Department of Pediatrics,Sun Yat-sen Memorial Hospital,Sun Yat-sen University,Guangzhou 510120,China)

机构地区：[1]中山大学孙逸仙纪念医院儿科,广州510120

出　　处：《新医学》2021年第8期637-640,共4页Journal of New Medicine

摘　　要：造血干细胞移植是目前唯一能治愈重型β地中海贫血的方法,但移植难度大,移植失败后再次移植风险更大,如何改进预处理方案提高移植成功率尚无定论,该文报道了1例接受非血缘HLA全相合供者造血干细胞移植术后发生原发排斥并接受二次移植成功患儿。该例患儿被确诊为Pesaron分度Ⅲ度的重型β地中海贫血,首次接受非血缘HLA全相合供者造血干细胞移植术,移植失败,原发排斥,自身造血恢复后1年再次接受另一非血缘HLA全相合供者外周血干细胞移植术。二次移植前30 d予氟达拉滨联合地塞米松免疫抑制治疗,调整预处理方案,并输注骨髓间充质干细胞促进植入。二次移植后患儿获得稳定植入,完全脱离输血状态至35个月,发生肺炎1次,无其他并发症。该例治疗结果提示,移植前予氟达拉滨联合地塞米松抑制受者T淋巴细胞功能可促进植入。减低强度预处理方案,辅助输注骨髓间充质干细胞促进造血重建,可以克服非血缘外周血干细胞移植治疗重型β地中海贫血的原发排斥,促进二次移植成功。Currently,hematopoietic stem cell transplantation(HSCT)is the only approach to treat severeβ-thalassemia.Nevertheless,this technology is highly challenging,and the risk of second transplantation is even higher after the failure of the first transplantation.How to improve the success rate of transplantation by modifying the pretreatment regime remains elusive.Here,we reported 1 child who successfully underwent the second HSCT after the failure of the first unrelated HSCT from a HLA-matched donor due to primary rejection.The child was diagnosed with severeβ-thalassemia,and untreated after the first unrelated HSCT from a HLA-matched donor due to primary rejection.At 1 year after the recovery of hematopoietic reconstitution,the second unrelated HSCT from another HLA-matched donor was performed.At preoperative 30 d,fludarabine combined with dexamethasone was given as the immunosuppressive therapy,the pretreatment regime was adjusted,and bone marrow mesenchymal stem cells were infused to promote the engraftment.After the second HSCT,the child achieved stable engraftment.From complete recovery of hematopoietic reconstitution until postoperative 35 months,the patient developed pneumonia once without other complications.This case prompts that suppressing T lymphocyte function of the recipients by fludarabine combined with dexamethasone before transplantation can promote the engraftment.Lowering the intensity of pretreatment regime and adjuvant infusion of bone marrow mesenchymal stem cells can promote hematopoietic reconstitution,resolve the primary rejection of unrelated HSCT from a HLA-matched donor and enhance the success of second HSCT.

关 键 词：地中海贫血 免疫治疗 造血干细胞移植 二次移植 

分 类 号：R457.7[医药卫生—治疗学] R556.61[医药卫生—临床医学]