Lin H C, Lin K H, Wang P J
Department of Pediatrics, En Chu Kong Hospital, San-Shia, Taipei, Taiwan, R.O.C.
Zhonghua Min Guo Xiao Er Ke Yi Xue Hui Za Zhi. 1998 Jul-Aug;39(4):260-3.
We report the result of allogeneic bone marrow transplantation (BMT) in a 14-year-old boy who was neurologically severely involved with the childhood form of adrenoleukodystrophy (ALD) and received marrow from his HLA-A and B nonidentical, MLC-nonreactive paternal donor without T-cell depletion processing. Bone marrow transplantation corrected the excess content of very long chain fatty acid in plasma but did not arrest the deterioration of the neurological status during 3.5-year post-transplant follow-up period. Since partially matched or unrelated donors have been applied to clinical BMT successfully with current new techniques, ALD patients will have a better prognosis when they are transplanted in status of mild and early involvement. Our first experience may be helpful in more trials of BMT for genetic leukodystrophy in Taiwan.
我们报告了一名14岁男孩接受异基因骨髓移植（BMT）的结果。该男孩患有儿童型肾上腺脑白质营养不良（ALD），神经系统严重受累，接受了来自其HLA - A和B不匹配、混合淋巴细胞培养无反应的父亲供体的骨髓，且未经过T细胞去除处理。骨髓移植纠正了血浆中极长链脂肪酸的过量含量，但在移植后3.5年的随访期内并未阻止神经状态的恶化。由于目前新技术已成功将部分匹配或无关供体应用于临床BMT，ALD患者在轻度和早期受累状态下进行移植时预后会更好。我们的首次经验可能有助于台湾地区开展更多针对遗传性脑白质营养不良的BMT试验。
