期刊
BONE MARROW TRANSPLANTATION
卷 41, 期 -, 页码 S87-S89出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/bmt.2008.62
关键词
haematopoietic cell transplantation; metabolic inborn errors; lysosomal diseases; mucopolysaccharidosis; leukodystrophy
Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.
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