The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation

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. Althou...

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Bibliographic Details
Published in:Biology of blood and marrow transplantation 2008-05, Vol.14 (5), p.485-498
Main Authors: Aldenhoven, Mieke, Boelens, JaapJan, de Koning, Tom J
Format: Article
Language:English
Subjects:
Central Nervous System Diseases - etiology
Central Nervous System Diseases - prevention & control
Clinical outcome
Hematology, Oncology and Palliative Medicine
Humans
Hurler Syndrome
Long-term outcome
Mucopolysaccharidosis I
Mucopolysaccharidosis I - complications
Mucopolysaccharidosis I - pathology
Mucopolysaccharidosis I - therapy
Stem cell transplantation
Stem Cell Transplantation - adverse effects
Stem Cell Transplantation - methods
Treatment Outcome
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Description
Summary: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 outlined, as well as the limitations of SCT in HS patients.
ISSN:1083-8791
1523-6536
DOI:10.1016/j.bbmt.2008.01.009