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The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation

Aldenhoven, Mieke ; Boelens, Jaap Jan and de Koning, Tom J. LU (2008) In Biology of Blood and Marrow Transplantation 14(5). p.485-498
Abstract

Hurler syndrome (HS) is a severe inborn error of metabolism causing progressive multi-system morbidity and death in early childhood. At present, stem cell transplantation (SCT) is the only available treatment that can prevent central nervous system disease progression in HS patients. Although SCT has been shown to be effective for several important clinical outcome parameters, the reported clinical outcome after successful SCT is variable among HS patients and there are still some major limitations. This review will focus on the clinical outcome of HS patients after successful SCT, with particular emphasis on the long-term outcome and complications. In addition, factors that are suggested to contribute to the variable outcome are... (More)

Hurler syndrome (HS) is a severe inborn error of metabolism causing progressive multi-system morbidity and death in early childhood. At present, stem cell transplantation (SCT) is the only available treatment that can prevent central nervous system disease progression in HS patients. Although SCT has been shown to be effective for several important clinical outcome parameters, the reported clinical outcome after successful SCT is variable among HS patients and there are still some major limitations. This review will focus on the clinical outcome of HS patients after successful SCT, with particular emphasis on the long-term outcome and complications. In addition, factors that are suggested to contribute to the variable outcome are outlined, as well as the limitations of SCT in HS patients.

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author
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Clinical outcome, Hurler Syndrome, Long-term outcome, Mucopolysaccharidosis I, Stem cell transplantation
in
Biology of Blood and Marrow Transplantation
volume
14
issue
5
pages
14 pages
publisher
Elsevier
external identifiers
  • scopus:41849119871
  • pmid:18410891
ISSN
1083-8791
DOI
10.1016/j.bbmt.2008.01.009
language
English
LU publication?
no
id
a6a14a6d-e906-4675-8978-196108920ba3
date added to LUP
2020-02-28 13:54:16
date last changed
2020-06-03 05:18:25
@article{a6a14a6d-e906-4675-8978-196108920ba3,
  abstract     = {<p>Hurler syndrome (HS) is a severe inborn error of metabolism causing progressive multi-system morbidity and death in early childhood. At present, stem cell transplantation (SCT) is the only available treatment that can prevent central nervous system disease progression in HS patients. Although SCT has been shown to be effective for several important clinical outcome parameters, the reported clinical outcome after successful SCT is variable among HS patients and there are still some major limitations. This review will focus on the clinical outcome of HS patients after successful SCT, with particular emphasis on the long-term outcome and complications. In addition, factors that are suggested to contribute to the variable outcome are outlined, as well as the limitations of SCT in HS patients.</p>},
  author       = {Aldenhoven, Mieke and Boelens, Jaap Jan and de Koning, Tom J.},
  issn         = {1083-8791},
  language     = {eng},
  month        = {05},
  number       = {5},
  pages        = {485--498},
  publisher    = {Elsevier},
  series       = {Biology of Blood and Marrow Transplantation},
  title        = {The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation},
  url          = {http://dx.doi.org/10.1016/j.bbmt.2008.01.009},
  doi          = {10.1016/j.bbmt.2008.01.009},
  volume       = {14},
  year         = {2008},
}