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Long-Term Outcomes of Liver Transplantation in Type 1 Gaucher Disease

Ayto, RM; Hughes, DA; Jeevaratnam, P; Rolles, K; Burroughs, AK; Mistry, PK; Mehta, AB; (2010) Long-Term Outcomes of Liver Transplantation in Type 1 Gaucher Disease. AM J TRANSPLANT , 10 (8) 1934 - 1939. 10.1111/j.1600-6143.2010.03168.x.

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Abstract

Gaucher disease (GD) is the most prevalent lysosomal storage disorder. Enzyme replacement therapy (ERT) has demonstrable efficacy in reversing clinical and pathological manifestations of GD. We report four patients with GD and severe hepatic impairment who were successfully treated by orthotopic liver transplantation. Liver failure resulted from GD in two patients and due to a comorbidity in two others (HCV and autoimmune chronic active hepatitis). Following successful liver transplantation, patients received long-term ERT. Liver transplantation is a life-saving treatment for end-stage liver disease in patients with Gaucher disease. All four patients have had excellent outcomes from liver transplantation for up to 10 years postprocedure with no evidence of Gaucher-related pathology in the graft.

Type: Article
Title: Long-Term Outcomes of Liver Transplantation in Type 1 Gaucher Disease
DOI: 10.1111/j.1600-6143.2010.03168.x
Keywords: Gaucher, liver, transplantation, ENZYME REPLACEMENT THERAPY, HEPATIC-FIBROSIS, ABNORMALITIES, ELEVATION, REGISTRY, CHILDREN
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inst for Liver and Digestive Hlth
URI: http://discovery.ucl.ac.uk/id/eprint/115111
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