Bone marrow transplantation for mucopolysaccharidosis type I: Experience of two British centres

被引：138

作者：

Vellodi, A

Young, EP

Cooper, A

Wraith, JE

Winchester, B

Meaney, C

Ramaswami, U

Will, A

机构：

[1] INST CHILD HLTH,DIV BIOCHEM & GENET,LONDON,ENGLAND

[2] ROYAL MANCHESTER CHILDRENS HOSP,WILLINK BIOCHEM GENET UNIT,MANCHESTER M27 1HA,LANCS,ENGLAND

[3] ROYAL MANCHESTER CHILDRENS HOSP,BONE MARROW TRANSPLANT UNIT,MANCHESTER M27 1HA,LANCS,ENGLAND

来源：

ARCHIVES OF DISEASE IN CHILDHOOD | 1997年 / 76卷 / 02期

关键词：

mucopolysaccharidosis type I; bone marrow transplantation;

D O I：

10.1136/adc.76.2.92

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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.

引用

页码：92 / 99

页数：8

共 23 条

[1] RAPID TYPING OF TANDEMLY REPEATED HYPERVARIABLE LOCI BY THE POLYMERASE CHAIN-REACTION - APPLICATION TO THE APOLIPOPROTEIN-B 3' HYPERVARIABLE REGION [J].