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Unfavorable outcome of hematopoietic stem cell transplantation in two siblings with Wolman disease due to graft failure and hepatic complications.

Abstract
Wolman disease (WD) is a fatal storage disease caused by the deficiency of the lysosomal enzyme acid lipase. Only 3 cases of successful treatment by hematopoietic stem cell transplantation (HSCT), are reported. We report a case of two brothers with WD who were treated with HSCT, but both died from hepatic complications. Together they exemplify the obstacles to successful HSCT in WD. We suggest that different strategies should be investigated in order to offer treatment for this disease.
AuthorsAsaf Yanir, Mahmud Abed Allatif, Michael Weintraub, Polina Stepensky
JournalMolecular genetics and metabolism (Mol Genet Metab) Vol. 109 Issue 2 Pg. 224-6 (Jun 2013) ISSN: 1096-7206 [Electronic] United States
PMID23583223 (Publication Type: Case Reports, Journal Article)
CopyrightCopyright © 2013 Elsevier Inc. All rights reserved.
Topics
  • Child
  • Fatal Outcome
  • Hematopoietic Stem Cell Transplantation (adverse effects)
  • Humans
  • Liver Diseases (diagnosis, etiology, therapy)
  • Male
  • Wolman Disease (complications, therapy)

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