Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

Emma A. Meagher, Prediman K. Shah, Daniel J. Rader, Dominique Larrey, Cesare R. Sirtori, Robert A. Hegele, Maurizio Averna, Daniel Gaudet, Dirk Blom, LeAnne T. Bloedon, Marina Cuchel, Hendrik du Toit Theron, Claudia Stefanutti, Giovanni Battista Vigna, Pamela Foulds

2017-07-17

SCID: 54.1/zch2fcps

Abstract (AI)

omozygous familial hypercholesterolemia is a genetic disorder characterized by low-density lipoprotein (LDL)-receptor dysfunction, markedly elevated levels of LDL-cholesterol (LDL-C) and premature atherosclerosis. Patients are often poorly responsive to conventional lipid-lowering therapies that upregulate LDL-receptor expression. In a pivotal 78-week openlabel trial, lomitapide, titrated to the maximal tolerable dose, decreased LDL-C by 50% at the end of the efficacy phase (week 26) in patients with homozygous familial hypercholesterolemia.

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DOI

10.1161/circulationaha.117.028208

Publication Date

2017-07-17

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Authors

Emma A. Meagher

Prediman K. Shah

Daniel J. Rader

Dominique Larrey

Cesare R. Sirtori

Robert A. Hegele

Maurizio Averna

Daniel Gaudet

Dirk Blom

LeAnne T. Bloedon

Marina Cuchel

Hendrik du Toit Theron

Claudia Stefanutti

Giovanni Battista Vigna

Pamela Foulds

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