Bone marrow transplantation for Fanconi anemia. Adjustment of the dose of cyclophosphamide for preconditioning.

The American journal of pediatric hematology/oncology Pub Date : 1993-11-01

M Yabe, H Yabe, M Matsuda, T Hinohara, Y Oh, K Hattori, K Ishikawa, T Ohshima, H Yamamoto, S Kato

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Abstract

Purpose: Five patients with Fanconi anemia have been treated by bone marrow transplantation.

Patients and methods: 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.

Results: 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.

Conclusions: These results indicate that the individual dose adjustment of CY used for preconditioning may prevent graft failure and severe acute GVHD.

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骨髓移植治疗范可尼贫血。环磷酰胺预处理剂量的调整。

目的:对5例范可尼贫血患者进行骨髓移植治疗。患者和方法:采用环磷酰胺(CY) (20 ~ 150 mg/kg)、抗淋巴细胞球蛋白、胸腹照射(4 ~ 6 Gy)。根据体外CY代谢物对范可尼贫血患者染色体的影响，单独调整CY预处理剂量。四名患者接受了来自人类白细胞抗原(HLA)相同的兄弟姐妹的骨髓，一名患者接受了来自其HLA表型相同的父亲的骨髓。结果:所有患者移植成功，未观察到急性移植物抗宿主病(GVHD) II级或以上。3例发展为慢性GVHD。所有患者移植后存活2-5年，血液学改善。结论:个体化调整CY预适应剂量可预防移植物衰竭和严重急性GVHD。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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The American journal of pediatric hematology/oncology

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