Vellodi A, Young E, Cooper A, Lidchi V, Winchester B, Wraith J E
Metabolic Unit, Great Ormond Street Hospital for Children NHS Trust, London, UK.
J Inherit Metab Dis. 1999 Jun;22(5):638-48. doi: 10.1023/a:1005525931994.
Bone marrow transplantation (BMT) was performed in 10 patients with Hunter disease (mucopolysaccharidosis type II, iduronate-2-sulphatase deficiency). The donor was an HLA-identical sibling in 2 cases, an HLA-nonidentical relative in 6 cases, a volunteer unrelated donor in 1 case, and details were not available in 1 case. Only three patients have survived for more than 7 years post BMT; however, this high mortality probably resulted from poor donor selection. In two, there has been a steady progression of physical disability and mental handicap. One patient has maintained normal intellectual development, with only mild physical disability. It is possible that BMT may be useful in selected patients with MPS II.
对10例亨特病（II型黏多糖贮积症，艾杜糖醛酸-2-硫酸酯酶缺乏症）患者进行了骨髓移植。2例供者为HLA全相合的同胞，6例为HLA不全相合的亲属，1例为志愿非血缘供者，1例供者情况不详。骨髓移植后仅3例患者存活超过7年；然而，这种高死亡率可能是由于供者选择不当所致。其中2例患者身体残疾和智力障碍持续进展。1例患者智力发育正常，仅存在轻度身体残疾。骨髓移植可能对部分II型黏多糖贮积症患者有用。
