Yabe M, Yabe H, Matsuda M, Hinohara T, Oh Y, Hattori K, Ishikawa K, Ohshima T, Yamamoto H, Kato S
Department of Pediatrics, Tokai University School of Medicine, Isehara, Japan.
Am J Pediatr Hematol Oncol. 1993 Nov;15(4):377-82.
Five patients with Fanconi anemia have been treated by bone marrow transplantation.
They were conditioned with cyclophosphamide (CY) (20-150 mg/kg), antilymphocyte globulin, and thoracoabdominal irradiation (4-6 Gy). The dose of CY for preconditioning was adjusted individually, based on the in vitro effect of CY metabolites on the chromosomes of patients with Fanconi anemia. Four patients received marrow from human leukocyte antigen (HLA)-identical siblings, and one received marrow from his HLA phenotypically identical father.
All patients achieved engraftment, and acute graft-versus-host disease (GVHD) grade II or more was not observed. Three developed chronic GVHD. All patients are surviving 2-5 years after grafting, with hematological improvement.
These results indicate that the individual dose adjustment of CY used for preconditioning may prevent graft failure and severe acute GVHD.
对5例范可尼贫血患者进行了骨髓移植治疗。
他们接受了环磷酰胺（CY）（20 - 150mg/kg）、抗淋巴细胞球蛋白及胸腹联合照射（4 - 6Gy）的预处理。预处理时CY的剂量根据CY代谢产物对范可尼贫血患者染色体的体外作用进行个体化调整。4例患者接受了来自人类白细胞抗原（HLA）相同同胞的骨髓，1例接受了来自其HLA表型相同父亲的骨髓。
所有患者均实现植入，未观察到II级或更严重的急性移植物抗宿主病（GVHD）。3例发生了慢性GVHD。所有患者移植后存活2 - 5年，血液学状况得到改善。
这些结果表明，预处理时CY的个体化剂量调整可能预防移植失败和严重的急性GVHD。
