Borker Anupama, Yu Lolie C
Department of Pediatric Hematology/Oncology, Children's Hospital of New Orleans and Louisiana State University, Health Science Center, 70118, USA.
Metab Brain Dis. 2002 Sep;17(3):139-42. doi: 10.1023/a:1019969820308.
Adrenoleukodystrophy is an inherited disease characterized by progressive neurodegeneration with rapid deterioration to a vegetative state. Of the treatment modalities tried to date, BMT is the only one with the potential for cure. However, the high rate of morbidity and mortality associated with allogeneic transplant makes it necessary to try out novel measures to improve the outcome in these patients. We report here a case where we used CD34+ stem cell selection for allogeneic unrelated-donor BMT in a patient with adrenoleukodystrophy.
肾上腺脑白质营养不良是一种遗传性疾病,其特征为进行性神经退行性变并迅速恶化为植物人状态。在迄今为止尝试过的治疗方式中,骨髓移植是唯一有可能治愈的方法。然而,异基因移植相关的高发病率和死亡率使得有必要尝试新的措施来改善这些患者的治疗效果。我们在此报告一例使用CD34+干细胞选择进行异基因非亲属供体骨髓移植治疗肾上腺脑白质营养不良患者的病例。