Journal article

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

MM Page, EI Ekinci, RM Jones, PW Angus, PJ Gow, RC O'Brien

Internal Medicine Journal | WILEY | Published : 2014

Abstract

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.

Grants

Awarded by NHMRC


Awarded by Health Resources and Services Administration


Funding Acknowledgements

E. I. Ekinci is supported by a NHMRC Early Career Fellowship: Part-time Health Professional Research Fellowship (#1054312). This work was supported in part by Health Resources and Services Administration contract 234-2005-37011C. The content is the responsibility of the authors alone and does not necessarily reflect the views or policies of the Department of Health and Human Services, nor does mention of trade names, commercial products or organisations imply endorsement by the U. S. Government.