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a Medical Unit,
Institute of Child Health, 30 Guilford Street, London WC1N 1EH, b Division of Biochemistry
and Genetics, Institute of Child Health, London, c Willink Biochemical
Genetics Unit, Royal Manchester Children's Hospital, d Bone Marrow Transplant Unit,
Royal Manchester Children's Hospital
Correspondence to: Dr Vellodi.
Accepted 24 July 1996
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.
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