异基因造血干细胞移植治疗重型β地中海贫血  

Allogeneic hematopoietic stem cell transplantation for β-thalassemia major

作　　者：李春富[1] 何岳林[1] 张玉明[1] 杨明[1] 吴学东[1] 白静 

机构地区：[1]第一军医大学附属南方医院儿科,广州510515

出　　处：《中华血液学杂志》2003年第4期174-176,共3页Chinese Journal of Hematology

摘　　要：目的　研究异基因造血干细胞移植治疗重型 β地中海贫血的临床效果。 方法　确诊为重型 β地中海贫血患儿 15例 ,年龄 1～ 10岁 (中位年龄 3.5岁 ) ,其中男 8例 ,女 7例 ;Pesaro中心地中海贫血分度Ⅰ～Ⅱ度 12例 ,Ⅲ度 3例。对低体重供者使用G CSF动员后采集骨髓 +外周血造血干细胞。HLA相合同胞骨髓移植 10例 ,外周血造血干细胞移植 2例 ,脐带血移植 3例。随访时间为 6～ 5 4个月。结果　 15例中 9例长期无病生存 ,8例HLA相合同胞骨髓移植Ⅰ～Ⅱ度患儿 7例无病生存 ;2例母亲供髓HLA不相合移植 ,1例无病生存 ,1例未植入。 2例外周血造血干细胞移植 ,其中 1例立即接受骨髓移植并获成功。 2例无关供者脐带血移植均未长期植入。 15例患者中发生Ⅰ度和Ⅲ度GVHD者分别为 3例和 1例 ;4例并发间质性肺炎 ;所有长期无病生存者无后期合并症。结论　Ⅰ～Ⅱ度 β地中海贫血患儿骨髓移植成功率较高 ( 87.5 %)。对低体重供者使用G CSF动员后采集骨髓 +外周血造血干细胞方法是确保植入的有效办法。但有增加急性和慢性GVHD和植入综合征的倾向。Objective To investigate the effect of hematopoietic stem cell transplantation(HSCT) for β-thalassemia major. Methods Fifteen β-thalassemia major patients with a median age of 3.5 years (range 1～10 years) received allogeneic HSCT. According to the Pesaro's classification for thalassemia, 12 patients were grade Ⅰ～Ⅱ, and 3 grade Ⅲ. The bone marrow transplantation(BMT) plus peripheral blood stem cell (PBSC) transplant mobilized by granulocyte colony-stimulating factor (G-CSF) was used when donor is low body-weight. Result Of the fifteen patients, nine were disease-free survival (DFS) at a median follow-up of 2.5 years (range 6～54 months). Of eight grade Ⅰ～Ⅱ patients received HLA identical sibling BMT, seven were DFS, and of two received HLA mismatched marrow from their mother, one DFS, another unengrafted. Two patients received unrelated cord blood HSCT were both unengrafted. Two patients received PBSC transplantation alone were not engrafted, but one of them soon received BMT from the same donor and was DFS. The incidences of grade Ⅰ～Ⅱ and grade Ⅲ acute graft-versus-host disease (aGVHD) were 20%(3/15) and 6.7%(1/15), respectively. Interstitial pneumonia occurred in 4/15 (26.7%) patients. There were no long-term complications in the survivors. Conclusion Grade Ⅰ～Ⅱ β-thalassemia major patients received HLA identical sibling BMT had higher DFS. It was propitious for engraftment to use BMT plus PBSC, but with a higher incidence of acute and chronic GVHD.

关 键 词：重型Β地中海贫血 异基因造血干细胞移植 骨髓移植 脐带血移植 疗效 并发症 

分 类 号：R556.61[医药卫生—血液循环系统疾病]