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Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S87-9. doi: 10.1038/bmt.2008.62.

The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update.

Author information

1
1BMT Unit, Paediatric Department of Milano-Bicocca University, San Gerardo Hospital, Monza, Italy. attilio.rovelli@pediatriamonza.it

Abstract

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.

PMID:
18545253
DOI:
10.1038/bmt.2008.62
[Indexed for MEDLINE]

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