Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
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DOI：
10.1182/blood-2007-03-079665
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发表时间：
2007-10-01
期刊：
影响因子：
20.3
通讯作者：
Gluckman, Eliane
中科院分区：
文献类型：
--
作者：
Bernaudin, Francoise;Socie, Gerard;Gluckman, Eliane
异基因造血干细胞移植（HSCT）是镰状细胞病（SCD）的唯一治愈性治疗方法;然而，其使用受到移植相关死亡率（TRM）风险的限制。在1988年11月至2004年12月期间，87例年龄在2至22岁之间的严重SCD患者在法国接受了移植。脑血管病变是移植的主要适应症（55例患者）。所有患者均在清髓性预处理方案（CR）后接受来自同胞供体的移植物。研究期间CR的唯一变化是1992年3月引入抗胸腺细胞球蛋白（ATG）。使用ATG前的排异率为22.6%，而使用ATG后的排异率为3%。中位随访时间为6年（范围：2.0 - 17.9年），总生存率和无事件生存率（EFS）分别为93.1%和86.1%。移植物抗宿主病（GVHD）是TRM的主要原因。重要的是，脐带血移植受者没有发生GVHD。在显著减少后未检测到新的缺血性病变。随着时间的推移，结果显著改善：2000年1月后接受移植的44例患者的EFS率为95.3%。这些结果表明，对于植入后卒中风险较高的SCD儿童，应考虑将ATG清髓预处理后的HLA相合同胞HSCT作为标准治疗，
Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for sickle cell disease (SCD); nevertheless, its use has been limited by the risk of transplantation-related mortality (TRM). Between November 1988 and December 2004, 87 consecutive patients with severe SCD ranging from 2 to 22 years of age received transplants in France. Cerebral vasculopathy was the principal indication for transplantation (55 patients). All the patients received grafts from a sibling donor after a myeloablative conditioning regimen (CR). The only change in the CR during the study period was the introduction of antithymocyte globulin (ATG) in March 1992. The rejection rate was 22.6% before the use of ATG but 3% thereafter. With a median follow-up of 6 years (range, 2.0 to 17.9 years), the overall and event-free survival (EFS) rates were 93.1% and 86.1%, respectively. Graft versus host disease (GVHD) was the main cause of TRM. Importantly, cord blood transplant recipients did not develop GVHD. No new ischemic lesions were detected after were significantly reduced. The outcome improved significantly with time: the EFS rate among the 44 patients receiving transplants after January 2000 was 95.3%. These results indicate that HLA-identical sibling HSCT after myeloablative conditioning with ATG should be considered as a standard of care for SCD children who are at high risk for stroke after engraftment, and cerebral velocities