Li Chun-fu, He Yue-lin, Zhang Yu-ming, Yang Ming, Wu Xue-dong, Bai Jing
Department of Pediatrics, Nanfang Hospital, Guangzhou 510515, China.
Zhonghua Xue Ye Xue Za Zhi. 2003 Apr;24(4):174-6.
To investigate the effect of hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major.
Fifteen beta-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.
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 not engrafted. Two patients received unrelated cord blood HSCT were both not engrafted. 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.
Grade I - II beta-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.
探讨造血干细胞移植（HSCT）治疗重型β地中海贫血的效果。
15例重型β地中海贫血患者，中位年龄3.5岁（范围1 - 10岁），接受了异基因HSCT。根据地中海贫血的佩萨罗分类，12例为Ⅰ - Ⅱ级，3例为Ⅲ级。当供体体重较低时，采用骨髓移植（BMT）加粒细胞集落刺激因子（G-CSF）动员的外周血干细胞（PBSC）移植。
15例患者中，9例在中位随访2.5年（范围6 - 54个月）时无病生存（DFS）。8例接受HLA相合同胞BMT的Ⅰ - Ⅱ级患者中，7例DFS；2例接受其母亲HLA不相合骨髓的患者中，1例DFS，另1例未植入。2例接受无关脐血HSCT的患者均未植入。2例单独接受PBSC移植的患者均未植入，但其中1例很快接受了同一供体的BMT并DFS。Ⅰ - Ⅱ级和Ⅲ级急性移植物抗宿主病（aGVHD）的发生率分别为20%（3/15）和6.7%（1/15）。4/15（26.7%）患者发生间质性肺炎。幸存者无长期并发症。
接受HLA相合同胞BMT的Ⅰ - Ⅱ级重型β地中海贫血患者DFS较高。采用BMT加PBSC有利于植入，但急慢性GVHD发生率较高。
