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

Asaf Yanir; Mahmud Abed Allatif; Michael Weintraub; Polina Stepensky

doi:10.1016/j.ymgme.2013.03.007

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

Mol Genet Metab. 2013 Jun;109(2):224-6. doi: 10.1016/j.ymgme.2013.03.007. Epub 2013 Mar 20.

Authors

Asaf Yanir¹, Mahmud Abed Allatif, Michael Weintraub, Polina Stepensky

Affiliation

¹ Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel. [email protected]

PMID: 23583223
DOI: 10.1016/j.ymgme.2013.03.007

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.

Publication types

Case Reports

MeSH terms

Child
Fatal Outcome
Hematopoietic Stem Cell Transplantation / adverse effects*
Humans
Liver Diseases / diagnosis*
Liver Diseases / etiology
Liver Diseases / therapy
Male
Wolman Disease / complications
Wolman Disease / therapy*