Gluckman E, Devergie A, Schaison G, Bussel A, Berger R, Sohier J, Bernard J
Br J Haematol. 1980 Aug;45(4):557-64. doi: 10.1111/j.1365-2141.1980.tb07178.x.
Five patients with Fanconi anaemia have been treated by bone marrow transplantation from HLA identical donors. Only one patient survived for more than 3 years. She is now perfectly healthy with complete haematological reconstitution with chimaerism and disparition of chromosomal abnormalities. In contrast, four patients died of acute severe GVHD soon after grafting. In addition, all had signs of severe cyclophosphamide toxicity. This evolution could be explained by a special sensitivity of FA cells to alkylating agents and may indicate the need to modify the conditioning regimen in FA patients.
五名范可尼贫血患者接受了来自 HLA 相同供体的骨髓移植治疗。只有一名患者存活超过 3 年。她现在完全健康，血液学完全重建，有嵌合体形成且染色体异常消失。相比之下，四名患者在移植后不久死于急性严重移植物抗宿主病（GVHD）。此外，所有患者都有严重的环磷酰胺毒性迹象。这种病情发展可以用范可尼贫血（FA）细胞对烷化剂的特殊敏感性来解释，这可能表明需要调整 FA 患者的预处理方案。
