A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia
A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia
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DOI：
10.1038/sj.bmt.1701016
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发表时间：
1997-12-01
影响因子：
4.8
通讯作者：
Nagler, A
中科院分区：
文献类型：
--
作者：
Kapelushnik, J;Or, R;Nagler, A
异基因骨髓移植(BMT)是治疗Fanconi‘s贫血(FA)的一种有效方法，但由于移植前常用的烷化剂和辐射的敏感性增加，死亡率和移植相关的并发症通常很高。氟达拉滨是一种嘌呤类似物，已被证明是治疗慢性淋巴细胞白血病患者的有效调节剂。我们报告了一例白血病转化伴血小板减少的儿童FA，20%的成髓细胞在接受氟达拉滨/ATG/环磷酰胺预适应后成功地接受了骨髓移植。该方案耐受性良好，没有观察到与移植相关的并发症，植入迅速。这名儿童目前在骨髓移植后10个月，临床状况良好，血细胞计数正常，100%嵌合体，没有移植物抗宿主病(GVHD)的迹象。我们认为，这种以氟达拉滨为基础的方案可能在标准FA患者的BMT和转化FA患者的条件化治疗中有效。
Allogeneic bone marrow transplantation (BMT) is an effective therapy for Fanconi's anemia (FA), However, mortality and transplant-related complications are usually high due to increased sensitivity to the alkylating agents and radiation commonly used for pre-transplant conditioning, Fludarabine monophosphate is a purine analogue that has been proven effective as a conditioning agent for chronic lymphocytic leukemia patients. We report a child with FA in leukemic transformation with thrombocytopenia and 20% myeloblasts who underwent successful BMT following conditioning with fludarabine/ATG/cyclophosphamide. The regimen was well tolerated, no transplant-related complications were observed, and engraftment was rapid. The child is currently 10 months post-BMT, in excellent clinical condition with a normal blood count, 100% chimerism and no sign of graft-versus-host disease (GVHD). We suggest that this fludarabine-based regimen may be effective in the conditioning of standard, as well as transforming, FA patients for BMT.