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

  • Published:
Journal of Inherited Metabolic Disease

Abstract

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.

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Authors and Affiliations

  1. Metabolic Unit, Great Ormond Street Hospital for Children NHS Trust, London, UK

    A. Vellodi

  2. Chemical Pathology, Royal Manchester Children's Hospital, Pendlebury, Manchester, UK

    E. Young

  3. Department of Psychological Medicine, Royal Manchester Children's Hospital, Pendlebury, Manchester, UK

    V. Lidchi & J. E. Wraith

  4. Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Pendlebury, Manchester, UK

    A. Cooper

  5. Biochemistry, Endocrinology and Metabolism Unit, Institute of Child Health, London, UK

    B. Winchester

Authors
  1. A. Vellodi
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  2. E. Young
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  3. A. Cooper
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  4. V. Lidchi
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  5. B. Winchester
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  6. J. E. Wraith
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Vellodi, A., Young, E., Cooper, A. et al. Long-term follow-up following bone marrow transplantation for Hunter disease. J Inherit Metab Dis 22, 638–648 (1999). https://doi.org/10.1023/A:1005525931994

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  • DOI: https://doi.org/10.1023/A:1005525931994

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