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|Title:||Long-term outcomes of liver transplantation for homozygous familial hypercholesterolaemia in Australia and New Zealand.||Austin Authors:||Page, Michael M;Hardikar, Winita;Alex, George;Bates, Sue;Srinivasan, Shubha;Stormon, Michael;Hall, Kat;Evans, Helen M;Johnston, Peter;Chen, John;Wigg, Alan;John, Libby;Ekinci, Elif I ;O'Brien, Richard C ;Jones, Robert M ;Watts, Gerald F||Affiliation:||Medical School, The University of Western Australia, Perth, Australia; Western Diagnostic Pathology, Perth, Australia.
Gastroenterology and Clinical Nutrition, The Royal Children's Hospital Melbourne, Melbourne, Australia; Department of Paediatrics, The University of Melbourne, Melbourne, Australia.
Institute of Endocrinology and Diabetes, The Children's Hospital at Westmead, Sydney, Australia; Faculty of Medicine and Health, University of Sydney, Sydney, Australia.
Faculty of Medicine and Health, University of Sydney, Sydney, Australia; Gastroenterology and Hepatology, The Children's Hospital at Westmead, Sydney, Australia.
Victorian Liver Transplant Unit
Paediatric Gastroenterology and Hepatology, Starship Child Health, Auckland, New Zealand; Faculty of Medical and Health Sciences, The University of Auckland, Auckland, New Zealand.
New Zealand Liver Transplant Unit, Auckland City Hospital, Auckland, New Zealand.
South Australia Liver Transplant Unit, Flinders Medical Centre, Adelaide, Australia;
College of Medicine and Public Health, Flinders University, Adelaide, Australia.
Medicine (University of Melbourne)
Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Melbourne, Australia.
Medical School, The University of Western Australia, Perth, Australia; Department of Cardiovascular Medicine, Royal Perth Hospital, Perth, Australia.
The Australian Centre for Accelerating Diabetes Innovation, Melbourne Medical School, Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Melbourne, Australia; Department of Medicine, Austin Health, Melbourne Medical School, The University of Melbourne, Melbourne, Australia.
|Issue Date:||10-Oct-2023||Date:||2023||Publication information:||Atherosclerosis 2023-10-10||Abstract:||Homozygous familial hypercholesterolaemia (FH) causes severe cardiovascular disease from childhood. Conventional drug therapy is usually ineffective; lipoprotein apheresis (LA) is often required. Liver transplantation (LT) can correct the metabolic defect but is considered a treatment of last resort. Newer drugs including lomitapide and evinacumab might reduce the need for apheresis and LT. We sought to determine the long-term outcomes following LT in Australia and New Zealand. We analysed demographic, biochemical and clinical data from all patients in Australia and New Zealand who have received LT for homozygous FH, identified from the Australia and New Zealand Liver and Intestinal Transplant Registry. Nine patients (five female; one deceased; seven aged between 3 and 6 years at the time of LT and two aged 22 and 26 years) were identified. Mean follow-up was 14.1 years (range 4-27). Baseline LDL-cholesterol off all treatment was 23 ± 4.1 mmol/L. Mean LDL-cholesterol on medical therapy (including maximal statin therapy in all patients, ezetimibe in three and LA in five) was 11 ± 5.7 mmol/L (p < 0.001). After LT, mean LDL-cholesterol was 2.6 ± 0.9 mmol/L (p = 0.004) with three patients remaining on statin therapy and none on LA. One patient died from acute myocardial infarction (AMI) three years after LT. Two patients required aortic valve replacement, more than 10 years after LT. The remaining six patients were asymptomatic after eight to 21 years of follow-up. No significant adverse events associated with immunosuppression were reported. LT for homozygous FH was highly effective in achieving substantial long-term reduction in LDL-cholesterol concentrations in all nine patients. LT remains an option for severe cases of homozygous FH where drug therapy combined with apheresis is ineffective or unfeasible.||URI:||https://ahro.austin.org.au/austinjspui/handle/1/34027||DOI:||10.1016/j.atherosclerosis.2023.117305||ORCID:||Journal:||Atherosclerosis||Start page:||117305||PubMed URL:||37863699||ISSN:||1879-1484||Type:||Journal Article|
|Appears in Collections:||Journal articles|
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