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Long‐term follow‐up following bone marrow transplantation for Hunter disease
Author(s) -
Vellodi A.,
Young E.,
Cooper A.,
Lidchi V.,
Winchester B.,
Wraith J. E.
Publication year - 1999
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1023/a:1005525931994
Subject(s) - sibling , hunter syndrome , medicine , mucopolysaccharidosis , bone marrow transplantation , mucopolysaccharidosis type ii , transplantation , disease , pediatrics , human leukocyte antigen , bone marrow , surgery , immunology , psychology , enzyme replacement therapy , developmental psychology , antigen
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.