HSCT for Fanconi anemia in children: factors that influence early and late results
HSCT for Fanconi anemia in children: factors that influence early and late results
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DOI：
10.1038/bmt.2008.284
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发表时间：
2008-10-01
影响因子：
4.8
通讯作者：
Dalle, J-H
中科院分区：
文献类型：
--
作者：
Dalle, J-H
Fanconi贫血(FA)是一种罕见的常染色体隐性遗传病，以先天畸形、癌症易感性和进行性骨髓衰竭为特征。FA患者存在自发和诱发的染色体断裂。造血干细胞移植(HSCT)是骨髓衰竭或克隆性造血异常时恢复正常造血的唯一治疗选择。传统的清髓预适应方案，特别是包括大剂量照射的方案，对FA患者似乎有很强的毒性。然后，针对这些患者成功地开发了降低强度的预适应方案。然而，TRM仍然高于其他HSCT指征。含有非清髓性预适应方案的氟达拉滨的开发似乎是一项重大进展。长期的跟踪是绝对必要的。
Fanconi anemia (FA) is a rare autosomal recessive disease characterized by congenital abnormalities, cancer predisposition and progressive BM failure. FA patients present spontaneous and induced chromosome breakage. Hematopoietic SCT (HSCT) represents the unique therapeutic option to restore normal hematopoiesis when marrow failure or clonal hematopoietic abnormality occurs. Conventional myeloablative conditioning regimen, especially including a high dose of irradiation, appeared strongly toxic for FA patients. Then, reduced-intensity conditioning regimens were developed successfully for those patients. However, TRM still remained higher than for other HSCT indications. The development of fludarabine containing a non-myeloablative conditioning regimen appears to be a major progress. Long-term follow-up is absolutely necessary.