title: 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.
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel. ayanir@hadassah.org.il
authors.forename: Asaf
authors.initials: A
authors.lastname: Yanir
authors.forename: Mahmud Abed
authors.initials: MA
authors.lastname: Allatif
authors.forename: Michael
authors.initials: M
authors.lastname: Weintraub
authors.forename: Polina
authors.initials: P
authors.lastname: Stepensky
mesh_terms: D002648:Child; D017809:Fatal Outcome; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D008107:Liver Diseases / Q000175:diagnosis* / Q000209:etiology / Q000628:therapy; D008297:Male; D015223:Wolman Disease / Q000150:complications / Q000628:therapy*