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

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  • A. Vellodi
    Metabolic Unit Great Ormond Street Hospital for Children NHS Trust London UK
  • E. Young
    Chemical Pathology Royal Manchester Children's Hospital, Pendlebury Manchester UK
  • A. Cooper
    Willink Biochemical Genetics Unit Royal Manchester Children's Hospital, Pendlebury Manchester UK
  • V. Lidchi
    Department of Psychological Medicine Royal Manchester Children's Hospital, Pendlebury Manchester UK
  • B. Winchester
    Biochemistry, Endocrinology and Metabolism Unit Institute of Child Health London UK
  • J. E. Wraith
    Department of Psychological Medicine Royal Manchester Children's Hospital, Pendlebury Manchester UK

Abstract

<jats:title>Abstract</jats:title><jats:p>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.</jats:p>

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