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

A Vellodi, E Young, A Cooper, V Lidchi… - Journal of inherited …, 1999 - Springer
A Vellodi, E Young, A Cooper, V Lidchi, B Winchester, JE Wraith
Journal of inherited metabolic disease, 1999Springer
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 …
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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