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重型再生障碍性贫血患儿的第二次异基因造血干细胞移植

Second allogeneic hematopoietic stem cell transplantation in children with severe aplastic anemia.

作者信息

Kudo K, Muramatsu H, Yoshida N, Kobayashi R, Yabe H, Tabuchi K, Kato K, Koh K, Takahashi Y, Hashii Y, Kawano Y, Inoue M, Cho Y, Sakamaki H, Kawa K, Kato K, Suzuki R, Kojima S

机构信息

Department of Pediatrics, Fujita Health University School of Medicine, Toyoake, Japan.

Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.

出版信息

Bone Marrow Transplant. 2015 Oct;50(10):1312-5. doi: 10.1038/bmt.2015.153. Epub 2015 Jun 29.

Abstract

The outcome of 55 children with severe aplastic anemia (SAA) who received a second hematopoietic stem cell transplantation (HSCT) was retrospectively analyzed using the registration data of the Japanese Society for Hematopoietic Cell Transplantation. The 5-year overall survival (OS) and failure-free survival (FFS) after the second transplantation were 82.9% (95% confidence interval (CI), 69.7-90.8)) and 81.2% (95% CI, 67.8-89.4), respectively. FFS was significantly better when the interval between the first and second transplantation was >60 days (88.9%; 95% CI, 73.0-95.7) than when it was ⩽60 days (61.4%; 95% CI, 33.3-80.5; P=0.026). All 12 patients who were conditioned with regimens containing fludarabine and melphalan were alive with hematopoietic recovery. These findings justify the recommendation of a second HSCT for children with SAA who have experienced graft failure after first HSCT.

摘要

利用日本造血细胞移植学会的登记数据,对55例接受第二次造血干细胞移植(HSCT)的重型再生障碍性贫血(SAA)患儿的结局进行了回顾性分析。第二次移植后的5年总生存率(OS)和无失败生存率(FFS)分别为82.9%(95%置信区间(CI),69.7 - 90.8))和81.2%(95% CI,67.8 - 89.4)。当第一次和第二次移植间隔>60天时,FFS显著优于间隔≤60天时(88.9%;95% CI,73.0 - 95.7)(61.4%;95% CI,33.3 - 80.5;P = 0.026)。所有12例接受含氟达拉滨和马法兰方案预处理的患者均存活且造血功能恢复。这些发现证明了对于首次HSCT后发生移植物失败的SAA患儿推荐进行第二次HSCT是合理的。

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