Ashizawa Masahiro, Akahoshi Yu, Nakano Hirofumi, Ugai Tomotaka, Wada Hidenori, Yamasaki Ryoko, Ishihara Yuko, Kawamura Koji, Sakamoto Kana, Sato Miki, Terasako Kiriko, Kimura Shun-Ichi, Kikuchi Misato, Nakasone Hideki, Kako Shinichi, Kanda Junya, Yamazaki Rie, Tanihara Aki, Nishida Junji, Kanda Yoshinobu
Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847 Amanuma, Omiya-ku, Saitama, Saitama, 330-8503, Japan.
Int J Hematol. 2014 Mar;99(3):311-7. doi: 10.1007/s12185-014-1501-6. Epub 2014 Feb 1.
Conditioning regimens consisting of reduced-dose cyclophosphamide (CY) and fludarabine (FDR) have been investigated for use in allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with aplastic anemia to reduce the toxicities associated with CY. However, the ideal dose of CY has not been identified. In addition, little information is available regarding donor cell chimerism after allo-HSCT with these regimens. Therefore, we retrospectively analyzed 13 patients who underwent allo-HSCT with half-dose CY (100 mg/kg in total), FDR, and anti-thymocyte globulin at total doses of 2.5-10 mg/kg at our center. All the patients except one, who died due to encephalopathy on day 20, achieved neutrophil engraftment a median of 18.5 days after HSCT with complete donor-type chimerism. Two patients who received a graft from an HLA-matched donor subsequently developed mixed chimerism (MC) associated with transfusion-dependent cytopenia. One became transfusion-independent after donor lymphocyte infusion, but continues to exhibit MC. The other regained complete donor-type chimerism after the cessation of cyclosporine, but remains transfusion-dependent. These findings suggest that a conditioning regimen with half-dose CY and FDR is effective for achieving neutrophil engraftment and complete donor-type chimerism. However, subsequent MC may be observed, especially after HLA-matched HSCT.
由低剂量环磷酰胺（CY）和氟达拉滨（FDR）组成的预处理方案已被研究用于再生障碍性贫血患者的异基因造血干细胞移植（allo-HSCT），以降低与CY相关的毒性。然而，CY的理想剂量尚未确定。此外，关于采用这些方案进行allo-HSCT后的供体细胞嵌合情况，可用信息很少。因此，我们回顾性分析了13例在本中心接受半量CY（总量100mg/kg）、FDR以及总量为2.5 - 10mg/kg抗胸腺细胞球蛋白进行allo-HSCT的患者。除1例在第20天因脑病死亡外，所有患者在HSCT后中位18.5天实现中性粒细胞植入，且为完全供者型嵌合。2例接受HLA匹配供者移植的患者随后出现与输血依赖血细胞减少相关的混合嵌合（MC）。其中1例在供者淋巴细胞输注后不再依赖输血，但仍表现为MC。另1例在停用环孢素后恢复完全供者型嵌合，但仍依赖输血。这些结果表明，半量CY和FDR的预处理方案对于实现中性粒细胞植入和完全供者型嵌合是有效的。然而，后续可能会观察到MC，尤其是在HLA匹配的HSCT后。
