I型黏多糖贮积症的骨髓移植：两个英国中心的经验
Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.
作者信息
Vellodi A, Young E P, Cooper A, Wraith J E, Winchester B, Meaney C, Ramaswami U, Will A
机构信息
Medical Unit, Institute of Child Health, London.
出版信息
Arch Dis Child. 1997 Feb;76(2):92-9. doi: 10.1136/adc.76.2.92.
Abstract
Bone marrow transplantation was carried out on 38 patients with mucopolysaccharidosis type I over a period of 15 years. The donor was an HLA identical relative in 10 cases, an HLA non-identical relative in 16 cases, and an HLA identical unrelated volunteer donor in 12 cases. Ten patients received a second transplant. One patient received three transplants. Thirteen engrafted patients have survived five years or more. Most patients have shown an arrest or slowing down of psychomotor regression. However, dysostosis multiplex has progressed. Careful selection of patients may be necessary to ensure optimum results.
摘要
在15年的时间里，对38例I型黏多糖贮积症患者进行了骨髓移植。供者为10例HLA相合的亲属、16例HLA不相合的亲属以及12例HLA相合的无关志愿供者。10例患者接受了第二次移植。1例患者接受了三次移植。13例植入成功的患者存活了5年或更长时间。大多数患者的精神运动发育迟缓已停止或减缓。然而，多发性骨发育异常仍在进展。可能需要仔细挑选患者以确保获得最佳结果。
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