Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy |
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| Authors: | Tyler Mark Pierson MD PhD Carsten G. Bonnemann MD Richard S. Finkel MD Nancy Bunin MD Gihan I. Tennekoon MD |
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| Affiliation: | 1. Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA;2. Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA |
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| Abstract: | Three siblings with metachromatic leukodystrophy underwent umbilical cord blood transplantation at different stages of disease. Neuroimaging, nerve conduction studies, neurological examinations, and neuropsychological examinations were used to measure outcome over 2 years. After transplant, the oldest sibling experienced disease progression. His two siblings had near or total resolution of signal abnormalities on neuroimaging. Their neuropsychological testing remained stable, and nerve conduction studies have shown improvement. These results indicate pretransplantation neurological examinations may be the most significant predictor of outcome after transplant. To our knowledge, this report is the first to document neurological outcome of metachromatic leukodystrophy treated by umbilical cord blood transplantation. Ann Neurol 2008;64:583–587 |
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