Transplantation for Adrenoleukodystrophy with HLA-A and B Nonidentical Paternal Marrow: Report of One Case

以人體白血球抗原不一致之父親骨髓移植於腎上腺腦白質退化不良症病人：一病例報告

林宏琪(Haung-Chi Lin) ； 林凱信(Kai-Hsin Lin) ； 王本榮(Pen-Jung Wang)

《Acta Paediatrica Sinica》 39卷4期 (1998/08) Pp. 260-263

https://doi.org/10.7097/APS.199808.0260

摘要

一名十四歲罹患腎上腺腦白質退化不良症男孩，於本院接受未經減去T型淋巴球處理之骨髓移植，以嘗試治療其嚴重神經症狀；骨髓來源爲人體白血球抗原不一致，混合淋巴培養不反應之其父。於移植後追蹤期間，我們發現骨髓移植雖然未能遏阻其神經症狀繼續惡化，但血漿中超長鏈脂肪酸過量情形確有改善。我們相信，在骨髓移植於臨床應用愈加精進的今日，若腎上腺腦白質病變病人能於受侵害之早期接受骨髓移植，其預後應可大爲改善。以爲台灣第一例以骨髓移植治療腎上腺腦白質退化不良症之報告。

關鍵字

腎上腺腦白質退化之良症 ； 人體白血球抗原 ； 骨髓移植

並列摘要

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.

並列關鍵字

adrenoleukodystrophy ； human leukocyte antigen ； bone marrow transplantation