PCSK9 Inhibitors for Homozygous Familial Hypercholesterolemia Useful But Seldom Sufficient

被引：9

作者：

Thompson, Gilbert R. ^{[1
]}

机构：

[1] Imperial Coll London, Fac Med, Hammersmith Hosp Campus, London, England

来源：

JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY | 2020年 / 76卷 / 02期

关键词：

alirocumab; apheresis; evolocumab; homozygous FH; lomitapide; PCSK9; inhibitor; OPEN-LABEL; EVOLOCUMAB; ATHEROSCLEROSIS; EFFICACY; THERAPY; SAFETY;

D O I：

10.1016/j.jacc.2020.05.033

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：143 / 145

页数：3

共 11 条

[1] Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia The ODYSSEY HoFH Trial [J].

Blom, Dirk J. ;

Harada-Shiba, Mariko ;

Rubba, Paolo ;

Gaudet, Daniel ;

Kastelein, John J. P. ;

Charng, Min-Ji ;

Pordy, Robert ;

Donahue, Stephen ;

Ali, Shazia ;

Dong, Yuping ;

Khilla, Nagwa ;

Banerjee, Poulabi ;

Baccara-Dinet, Marie ;

Rosenson, Robert S. .

JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2020, 76 (02) :131-142

[2] FAMILIAL HYPERCHOLESTEROLEMIA - DEFECTIVE BINDING OF LIPOPROTEINS TO CULTURED FIBROBLASTS ASSOCIATED WITH IMPAIRED REGULATION OF 3-HYDROXY-3-METHYLGLUTARYL COENZYME A REDUCTASE-ACTIVITY [J].

BROWN, MS ;

GOLDSTEIN, JL .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1974, 71 (03) :788-792

[3] Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society [J].

Cuchel, Marina ;

Bruckert, Eric ;

Ginsberg, Henry N. ;

Raal, Frederick J. ;

Santos, Raul D. ;

Hegele, Robert A. ;

Kuivenhoven, Jan Albert ;

Nordestgaard, Borge G. ;

Descamps, Olivier S. ;

Steinhagen-Thiessen, Elisabeth ;

Tybjrg-Hansen, Anne ;

Watts, Gerald F. ;

Averna, Maurizio ;

Boileau, Catherine ;

Boren, Jan ;

Catapano, Alberico L. ;

Defesche, Joep C. ;

Hovingh, G. Kees ;

Humphries, Steve E. ;

Kovanen, Petri T. ;

Masana, Luis ;

Pajukanta, Paivi ;

Parhofer, Klaus G. ;

Ray, Kausik K. ;

Stalenhoef, Anton F. H. ;

Stroes, Erik ;

Taskinen, Marja-Riitta ;

Wiegman, Albert ;

Wiklund, Olov ;

Chapman, M. John .

EUROPEAN HEART JOURNAL, 2014, 35 (32) :2146-U100

[4] Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study [J].

Cuchel, Marina ;

Meagher, Emma A. ;

Theron, Hendrik du Toit ;

Blom, Dirk J. ;

Marais, A. David ;

Hegele, Robert A. ;

Averna, Maurizio R. ;

Sirtori, Cesare R. ;

Shah, Prediman K. ;

Gaudet, Daniel ;

Stefanutti, Claudia ;

Vigna, Giovanni B. ;

Du Plessis, Anna M. E. ;

Propert, Kathleen J. ;

Sasiela, William J. ;

Bloedon, LeAnne T. ;

Rader, Daniel J. .

LANCET, 2013, 381 (9860) :40-46

[5] FAMILIAL HYPERCHOLESTEROLEMIA - IDENTIFICATION OF A DEFECT IN REGULATION OF 3-HYDROXY-3-METHYLGLUTARYL COENZYME-A REDUCTASE-ACTIVITY ASSOCIATED WITH OVERPRODUCTION OF CHOLESTEROL [J].

GOLDSTEIN, JL ;

BROWN, MS .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1973, 70 (10) :2804-2808

[6] Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study [J].

Raal, Frederick J. ;

Hovingh, G. Kees ;

Blom, Dirk ;

Santos, Raul D. ;

Harada-Shiba, Mariko ;

Bruckert, Eric ;

Couture, Patrick ;

Soran, Handrean ;

Watts, Gerald F. ;

Kurtz, Christopher ;

Honarpour, Narimon ;

Tang, Lihua ;

Kasichayanula, Sree ;

Wasserman, Scott M. ;

Stein, Evan A. .

LANCET DIABETES & ENDOCRINOLOGY, 2017, 5 (04) :280-290

[7] PCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trial [J].

Raal, Frederick J. ;

Stein, Evan A. ;

Dufour, Robert ;

Turner, Traci ;

Civeira, Fernando ;

Burgess, Lesley ;

Langslet, Gisle ;

Scott, Russell ;

Olsson, Anders G. ;

Sullivan, David ;

Hovingh, G. Kees ;

Cariou, Bertrand ;

Gouni-Berthold, Ioanna ;

Somaratne, Ransi ;

Bridges, Ian ;

Scott, Rob ;

Wasserman, Scott M. ;

Gaudet, Daniel .

LANCET, 2015, 385 (9965) :331-340

[8] Efficacy and Safety of Evolocumab in Reducing Lipids and Cardiovascular Events [J].

Sabatine, Marc S. ;

Giugliano, Robert P. ;

Wiviott, Stephen D. ;

Raal, Frederick J. ;

Blom, Dirk J. ;

Robinson, Jennifer ;

Ballantyne, Christie M. ;

Somaratne, Ransi ;

Legg, Jason ;

Wasserman, Scott M. ;

Scott, Robert ;

Koren, Michael J. ;

Stein, Evan A. .

NEW ENGLAND JOURNAL OF MEDICINE, 2015, 372 (16) :1500-1509

[9] Lipoprotein Apheresis in the Management of Familial Hypercholesterolaemia: Historical Perspective and Recent Advances [J].

Stefanutti, Claudia ;

Thompson, Gilbert R. .

CURRENT ATHEROSCLEROSIS REPORTS, 2015, 17 (01)

[10] Atherosclerosis in cholesterol-fed rabbits and in homozygous and heterozygous LDL receptor-deficient humans [J].

Thompson, Gilbert R. .

ATHEROSCLEROSIS, 2018, 276 :148-154

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