Liver transplantation for the treatment of homozygous familial hypercholesterolaemia in an era of emerging lipid-lowering therapies

被引：28
作者：
Page, M. M. [1 ]
Ekinci, E. I. [2 ,4 ,5 ]
Jones, R. M. [3 ,4 ]
Angus, P. W. [3 ,4 ]
Gow, P. J. [3 ,4 ]
O'Brien, R. C. [2 ,4 ]
机构：
[1] Royal Perth Hosp, Lipid Disorders Clin, Perth, WA 6000, Australia
[2] Austin Hlth, Austin Hlth Endocrine Ctr, Melbourne, Vic, Australia
[3] Austin Hlth, Liver Transplant Unit, Melbourne, Vic, Australia
[4] Univ Melbourne, Dept Med, Melbourne, Vic, Australia
[5] Menzies Sch Hlth Res, Darwin, NT, Australia
来源：
INTERNAL MEDICINE JOURNAL | 2014年 / 44卷 / 06期
基金：
澳大利亚国家健康与医学研究理事会;
关键词：
familial hypercholesterolaemia; liver transplantation; LDL-apheresis; mipomersen; lomitapide; HEART; LIPOPROTEINS; SURGERY; PATIENT; BYPASS;
D O I：
10.1111/imj.12444
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Homozygous familial hypercholesterolaemia (FH) causes severe premature coronary artery disease because of very high levels of low density lipoprotein (LDL)-cholesterol. Standard lipid-lowering drugs and LDL-apheresis may not be sufficiently effective. Liver transplantation replaces defective LDL receptors and vastly improves the lipid profile, and we present the first report of an Australian adult to receive this treatment. Emerging drug treatments for FH may be alternatives to LDL-apheresis and transplantation, but long-term safety and efficacy data are lacking for all of these options.
引用
收藏
页码：601 / 604
页数：4
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