Yabe Miharu, Shimizu Takashi, Morimoto Tsuyoshi, Koike Takashi, Takakura Hiromitsu, Tsukamoto Hideo, Muroi Kazuo, Oshima Koichi, Asami Keiko, Takata Minoru, Yamashita Takayuki, Kato Shunichi, Yabe Hiromasa
Department of Cell Transplantation, Tokai University Hospital, Kanagawa, Japan.
Pediatr Transplant. 2012 Jun;16(4):340-5. doi: 10.1111/j.1399-3046.2012.01669.x. Epub 2012 Mar 8.
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/m(2) of 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.
如果在骨髓增生异常综合征（MDS）或白血病发生之前进行，来自人类白细胞抗原（HLA）相同同胞供者的造血干细胞移植（SCT）通常与范可尼贫血（FA）患者的良好生存率相关。然而，最佳预处理方案尚未确定。我们在此报告15例接受HLA匹配同胞供者SCT的日本FA患者的经验。本研究的目的是在日本人群（其中T细胞体细胞镶嵌现象高于白种人群）中比较基于放疗的预处理与基于氟达拉滨（Flu）的预处理对FA患者的效果。8例患者（A组）接受基于放疗的预处理（胸腹/全身照射500 - 600 cGy）并调整环磷酰胺（CY）剂量（20 - 120 mg/kg）以及抗胸腺细胞球蛋白（ATG）；2例患者出现排斥反应。7例患者（B组）接受CY（40 mg/kg）、150 - 180 mg/m²的Flu以及ATG。所有患者均实现了持久植入。对于FA患者，基于Flu的预处理即使在存在T细胞体细胞镶嵌现象的患者中，也可能在不进行放疗的情况下使匹配同胞供者移植实现稳定植入。
