Long-term follow-up following bone marrow transplantation for Hunter disease

被引：124

作者：

Vellodi, A

Young, E

Cooper, A

Lidchi, V

Winchester, B

Wraith, JE

机构：

[1] Great Ormond St Hosp Sick Children, Metab Unit, London WC1N 3JH, England

[2] Royal Manchester Childrens Hosp, Dept Psychol Med, Manchester M27 1HA, Lancs, England

[3] Royal Manchester Childrens Hosp, Willink Biochem Genet Unit, Manchester M27 1HA, Lancs, England

[4] Inst Child Hlth, Biochem Endocrinol & Metab Unit, London, England

来源：

JOURNAL OF INHERITED METABOLIC DISEASE | 1999年 / 22卷 / 05期

关键词：

D O I：

10.1023/A:1005525931994

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：638 / 648

页数：11

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