Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
3
pubmed:dateCreated
2004-6-3
pubmed:abstractText
The only effective treatment for selected metabolic diseases is a successful allogeneic hematopoietic stem cell transplantation (HCT). Best results with HCT are obtained when performed early in the course of the disease. Hence, timely identification and referral are critical. Also, early identification of affected patients during the newborn period via screening may be invaluable, particularly for the infantile onset rapidly progressive forms of diseases. Rapid availability of a donor graft is often crucial for these patients. Preliminary experience suggests that results after umbilical cord blood (UCB) transplant may be comparable to those after marrow transplants. UCB grafts have certain benefits of rapid availability and even reduced risk of GvHD. Hence, UCB transplant represents an alternative to marrow HCT. Related haploidentical HCT, possibly with very high doses of CD34+ cells, may also represent an option. However, expertise has been developed in very few transplant centers and no large reports are available of its use for patients with inherited metabolic diseases.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:status
MEDLINE
pubmed:issn
0300-8630
pubmed:author
pubmed:issnType
Print
pubmed:volume
216
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
163-8
pubmed:dateRevised
2005-11-16
pubmed:meshHeading
pubmed:articleTitle
Hematopoietic stem cell transplantation for mucopolysaccharidoses and leukodystrophies.
pubmed:affiliation
Division of Pediatric Hematology/Oncology, Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA. sauer.martin@mh-hannover.de
pubmed:publicationType
Journal Article, Review