HEART UK statement on the management of homozygous familial hypercholesterolaemia in the United Kingdom

被引：84

作者：

France, Michael ^{[1
]}

Rees, Alan ^{[2
]}

Datta, Dev ^{[3
]}

Thompson, Gilbert ^{[4
]}

Capps, Nigel ^{[5
]}

Ferns, Gordon ^{[6
]}

Ramaswami, Uma ^{[7
]}

Seed, Mary ^{[8
]}

Neely, Dermot ^{[9
]}

Cramb, Robert ^{[10
]}

Shoulders, Carol ^{[11
]}

Barbir, Mahmoud ^{[12
]}

Pottle, Alison ^{[13
]}

Eatough, Ruth ^{[14
]}

Martin, Steven ^{[15
]}

Bayly, Graham ^{[16
]}

Simpson, Bill ^{[17
]}

Halcox, Julian ^{[18
]}

Edwards, Ray ^{[2
]}

Main, Linda ^{[19
]}

Payne, Jules ^{[2
]}

Soran, Handrean ^{[20
,21
,22
]}

机构：

[1] Cent Manchester Univ Hosp, NHS Fdn Trust, Dept Clin Biochem, Oxford Rd, Manchester M13 9WL, Lancs, England

[2] HEART UK, 7 North Rd, Maidenhead SL6 1PE, Berks, England

[3] Univ Hosp, Lipid Unit, Penlan Rd, Cardiff CF62 2XX, S Glam, Wales

[4] Imperial Coll London, Hammersmith Hosp, Clin Lipidol, Du Cane Rd, London W12 ONN, England

[5] Princess Royal Hosp, Dept Clin Biochem, Telford TF1 6TF, Shrops, England

[6] Univ Sussex, Brighton & Sussex Med Sch, Kent Surrey & Sussex Clin Res Network, Div Med Educ, Brighton BN1 9PX, E Sussex, England

[7] Royal Free Hosp, Inst Immun & Transplantat, Lysosomal Disorders Unit, 2nd Floor, London NW3 2QG, England

[8] Imperial Coll Healthcare NHS Trust, Charing Cross Hosp, Dept Cardiol, Fulham Palace Rd, London W6 8RF, England

[9] Newcastle Tyne Hosp NHS Fdn Trust, Dept Blood Sci, Newcastle Upon Tyne NE1 4LP, Tyne & Wear, England

[10] Univ Hosp Birmingham, NHS Fdn Trust, Mindelsohn Way, Birmingham B15 2GW, W Midlands, England

[11] Queen Mary Univ, William Harvey Res Inst, Charterhouse Sq, London EC1M 6BQ, England

[12] NHLI Imperial Coll, Royal Brompton & Harefield NHS Fdn Trust, Hill End Rd, Harefield UB9 6JH, Middx, England

[13] Royal Brompton & Harefield NHS Fdn Trust, Hill End Rd, Harefield UB9 6JH, Middx, England

[14] Cent Manchester Univ Hosp, NHS Fdn Trust, Dept Med, Oxford Rd, Manchester M13 9WL, Lancs, England

[15] Peterborough City Hosp, NHS Fdn Trust, Peterborough & Stamford Hosp, Peterborough PE3 9GZ, England

[16] Univ Hosp Bristol NHS Fdn Trust, Bristol BS2 8HW, Avon, England

[17] Aberdeen Royal Infirm, Forester Hill, Aberdeen AB25 2ZN, Scotland

[18] Swansea Univ Prifysgol Abertawe, Coll Med, Swansea SA2 8PP, W Glam, Wales

[19] Drummond Consultants Ltd, 3A Bowmans Close, Burnham SL1 8LH, Bucks, England

[20] Cent Manchester Univ Hosp NHS Fdn Trust, Dept Med, Manchester M13 9WL, Lancs, England

[21] Cent Manchester Univ Hosp NHS Fdn Trust, Cardiovasc Trials Unit, Manchester M13 9WL, Lancs, England

[22] Univ Manchester, Cardiovasc Res Grp, Manchester M13 9WL, Lancs, England

来源：

ATHEROSCLEROSIS | 2016年 / 255卷

关键词：

Homozygous familial; hypercholesterolaemia; Lipoprotein apheresis; Lomitapide; evolocumab; proprotein convertase; subtilisin kexin type 9; Consensus; HEART UK; LOW-DENSITY-LIPOPROTEIN; NON-HDL-CHOLESTEROL; DONOR LIVER-TRANSPLANTATION; LDL-APHERESIS; CARDIOVASCULAR RISK; LIVING-DONOR; FOLLOW-UP; ANTITHROMBOTIC THERAPY; PCSK9; INHIBITORS; DOUBLE-BLIND;

D O I：

10.1016/j.atherosclerosis.2016.10.017

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

This consensus statement addresses the current three main modalities of treatment of homozygous familial hypercholesterolaemia (HoFH): pharmacotherapy, lipoprotein (Lp) apheresis and liver transplantation. HoFH may cause very premature atheromatous arterial disease and death, despite treatment with Lp apheresis combined with statin, ezetimibe and bile acid sequestrants. Two new classes of drug, effective in lowering cholesterol in HoFH, are now licensed in the United Kingdom. Lomitapide is restricted to use in HoFH but, may cause fatty liver and is very expensive. PCSK9 inhibitors are quite effective in receptor defective HoFH, are safe and are less expensive. Lower treatment targets for lipid lowering in HoFH, in line with those for the general FH population, have been proposed to improve cardiovascular outcomes. HEART UK presents a strategy combining Lp apheresis with pharmacological treatment to achieve these targets in the United Kingdom (UK). Improved provision of Lp apheresis by use of existing infrastructure for extracorporeal treatments such as renal dialysis is promoted. The clinical management of adults and children with HoFH including advice on pregnancy and contraception are addressed. A premise of the HEART UK strategy is that the risk of early use of drug treatments beyond their licensed age restriction may be balanced against risks of liver transplantation or ineffective treatment in severely affected patients. This may be of interest beyond the UK. Crown Copyright (C) 2016 Published by Elsevier Ireland Ltd.

引用

页码：128 / 139

页数：12

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