Matched sibling donor stem cell transplantation for Fanconi anemia patients with T-cell somatic mosaicism
Matched sibling donor stem cell transplantation for Fanconi anemia patients with T-cell somatic mosaicism
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匹配同胞供体干细胞移植治疗 T 细胞体细胞嵌合的范可尼贫血患者
DOI：
10.1111/j.1399-3046.2012.01669.x
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发表时间：
2012
期刊：
影响因子：
--
通讯作者：
Yabe H
中科院分区：
文献类型：
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作者：
Yabe M;Shimizu T;Morimoto T;Koike T;Takakura H;Tsukamoto H;Muroi K;Oshima K;Asami K;Takata M;Yamashita T;Kato S;Yabe H
Yabe M，Shimizu T，Morimoto T，Koike T，Takakura H，Tsukamoto H，Muroi K，Oshima K，Asami K，Takata M，Yamashita T，Kato S，Yabe H匹配同胞干细胞移植治疗T细胞体嵌合性Fanconi贫血患者。然而，最佳的调理方案尚未确定。我们在此报告15例日本FA患者的经验，他们接受了人类白细胞抗原相合的同胞供者SCT。这项研究的目的是在T细胞体嵌合性高于高加索人群的日本人群中，比较基于辐射的调理和基于流感的调理对FA患者的影响。8例患者(A组)接受基于辐射的预适应(5 0 0~6 0 0 cGy胸腹/脑外伤)加环磷酰胺(CY)剂量调整(2 0~12 0 mg/kg)，2例出现排斥反应。B组7例，CY 40 mg/kg，Flu 150~180 mg/m2，ATG。所有患者均证实了耐久植骨。在FA患者中，基于Flu的条件作用可能允许匹配的同胞供者移植中稳定的植入，而不需要辐射，即使在T细胞体细胞嵌合体患者中也是如此。
Yabe M, Shimizu T, Morimoto T, Koike T, Takakura H, Tsukamoto H, Muroi K, Oshima K, Asami K, Takata M, Yamashita T, Kato S, Yabe H. Matched sibling donor stem cell transplantation for Fanconi anemia patients with T‐cell somatic mosaicism.Abstract:SCT from HLA‐identical sibling donors is generally associated with an excellent survival in FA patients if performed prior to the development of MDS or leukemia. However, the optimal conditioning regimen has not been defined. We report here our experience with 15 Japanese FA patients who underwent HLA‐matched sibling donor SCT. The aim of this study is to compare radiation‐based conditioning to Flu‐based conditioning for FA patients in a Japanese population where the T‐cell somatic mosaicism is higher than in the Caucasian population. Eight patients (a‐group) received a radiation‐based conditioning (500–600 cGy of thoracoabdominal/TBI) with CY dose modification (20–120 mg/kg), and ATG; two patients exhibited rejection. Seven patients (b‐group) received CY (40 mg/kg), 150–180 mg/m2of Flu, and ATG. Durable engraftment was demonstrated in all patients. In FA patients, Flu‐based conditioning may allow stable engraftment in matched sibling donor transplantation without radiation, even in patients with T‐cell somatic mosaicism.