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Erschienen in: European Journal of Pediatrics 7/2007

01.07.2007 | Original Paper

Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation

verfasst von: Jerry Stein, Ben Zion Garty, Yael Dror, Eyal Fenig, Marsha Zeigler, Isaac Yaniv

Erschienen in: European Journal of Pediatrics | Ausgabe 7/2007

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Abstract

Wolman disease is a rapidly fatal lysosomal storage disease caused by the complete absence of lysosomal acid lipase activity. We report the cure of an infant with Wolman disease following transplantation of unrelated HLA-mismatched umbilical cord blood-derived stem cells. Umbilical cord blood was chosen as the stem-cell source because of its immediate availability and reduced tendency to cause graft-versus-host disease. The transplantation resulted in restoration of normal acid lipase levels before the onset of permanent end-organ damage. Four years after transplantation, the patient is thriving and has normal levels of acid lipase in peripheral blood cells. To our knowledge, this is the first report of a successful unrelated cord blood transplant in a patient with Wolman disease. Umbilical cord stem cells transplantation can restore acid lipase levels in Wolman disease, and if performed early, can cure the disease.
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Metadaten
Titel
Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation
verfasst von
Jerry Stein
Ben Zion Garty
Yael Dror
Eyal Fenig
Marsha Zeigler
Isaac Yaniv
Publikationsdatum
01.07.2007
Verlag
Springer-Verlag
Erschienen in
European Journal of Pediatrics / Ausgabe 7/2007
Print ISSN: 0340-6199
Elektronische ISSN: 1432-1076
DOI
https://doi.org/10.1007/s00431-006-0298-6

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