The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation
(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.
(Less)
- author
- Aldenhoven, Mieke ; Boelens, Jaap Jan and de Koning, Tom J. LU
- publishing date
- 2008-05-01
- 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
-
- pmid:18410891
- scopus:41849119871
- 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
- 2024-06-13 13:45:43
@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}},
keywords = {{Clinical outcome; Hurler Syndrome; Long-term outcome; Mucopolysaccharidosis I; Stem cell transplantation}},
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}},
}