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| Bone marrow transplantation for mucopolysaccharidosis type I: Experience of two British centres |
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| Author(s): Vellodi A, Young EP, Cooper A, Wraith JE, Winchester B, Meaney C, Ramaswami U, Will A |
| Source: ARCHIVES OF DISEASE IN CHILDHOOD Volume: 76 Issue: 2 Pages: 92-99 Published: FEB 1997 |
| Times Cited: 66 References: 23 |
| 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. |
| Document Type: Article |
| Language: English |
| Reprint Address: Vellodi, A (reprint author), INST CHILD HLTH, MED UNIT, 30 GUILFORD ST, LONDON WC1N 1EH, ENGLAND |
Addresses:
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 |
| Publisher: BRITISH MED JOURNAL PUBL GROUP, BRITISH MED ASSOC HOUSE, TAVISTOCK SQUARE, LONDON, ENGLAND WC1H 9JR |
| Subject Category: Pediatrics |
| IDS Number: WJ012 |
| ISSN: 0003-9888 |
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| | | | | | | | | Record from Web of Science® | | | | | | | | | |