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

被引：20

作者：

Ayto, R. M. ^{[1
]}

Hughes, D. A. ^{[1
]}

Jeevaratnam, P. ^{[1
]}

Rolles, K. ^{[2
,3
]}

Burroughs, A. K. ^{[2
,3
]}

Mistry, P. K. ^{[4
]}

Mehta, A. B. ^{[1
]}

Pastores, G. M. ^{[5
,6
]}

机构：

[1] Univ London Royal Free Hosp, Dept Haematol, Lysosomal Storage Disorders Unit, London NW3 2QG, England

[2] Univ London Royal Free Hosp, Royal Free Sheila Sherlock Liver Ctr, London NW3 2QG, England

[3] Univ London Royal Free Hosp, Univ Dept Surg, London NW3 2QG, England

[4] Yale Univ, Sch Med, Dept Pediat Gastroenterol & Hepatol, New Haven, CT USA

[5] NYU, Dept Neurol, Sch Med, New York, NY 10016 USA

[6] NYU, Dept Pediat, Sch Med, New York, NY 10016 USA

来源：

AMERICAN JOURNAL OF TRANSPLANTATION | 2010年 / 10卷 / 08期

关键词：

Gaucher; liver; transplantation; ENZYME REPLACEMENT THERAPY; HEPATIC-FIBROSIS; ABNORMALITIES; ELEVATION; REGISTRY; CHILDREN;

D O I：

10.1111/j.1600-6143.2010.03168.x

中图分类号：

R61 [外科手术学];

学科分类号：

摘要：

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.

引用

页码：1934 / 1939

页数：6

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