For patients with homozygous familial hypercholesterolemia, adding lomitapide — a microsomal triglyceride transfer protein inhibitor — reduced low-density lipoprotein cholesterol (LDL-C), with some patients reaching recommended LDL-C targets, according to a study published in the European Journal of Preventive Cardiology.

The aim of this study was to evaluate the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide at reducing LDL-C in patients with homozygous familial hypercholesterolemia who were receiving lipoprotein apheresis without reaching treatment targets. A clinical or genetic diagnosis of homozygous familial hypercholesterolemia was based on either the American Heart Association or the European Atherosclerosis Society statements. The Direct Adsorption of Lipoproteins system was used if, with diet and lipid-lowering medications, target LDL-C levels were still not being met. Lipid profiles were completed before and after each session, and carotid ultrasounds were completed before and after lomitapide treatment.

Of the 12 patients included in this study, 8 were men. Ages ranged between 8 and 62 years, and the median body mass index was 22.2 kg/m². The median range of LDL-C was 900 mg/dL before intervention, 383.5 mg/dL after lipid-lowering medication, 288.1 mg/dL after lipid-lowering medications and lipoprotein apheresis, and 173.5 mg/dL after lipid-lowering medications and lomitapide. Adding lomitapide to treatment lowered LDL-C by 56.8% compared with the administration of lipid-lowering medications only (P =.005), and by 54% when compared with lipid-lowering medication plus lipoprotein apheresis (P =.031). During the course of the study, 8.3% of the patients reduced LDL-C levels to <70 mg/dL; 25% of the patients reduced LDL-C levels to <100 mg/dL, and 42% of the patients reduced LDL-C levels to <130 mg/dL. Side effects were reported in 16.6% of patients and included transient diarrhea and liver transaminase >5 times the upper limit of normal.

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Limitations of this study included using calculated, rather than directly measured, LDL-C levels and not including a control cohort.

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The researchers concluded that “patients with [homozygous familial hypercholesterolemia] treated with maximum tolerated doses of [lipid-lowering] drugs and [lipoprotein apheresis] who had not achieved LDL-C treatment goals recommended by guidelines can be satisfactorily treated with lomitapide.”

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.


Kolovou G, Diakoumakou O, Kolovou V, et al. Microsomal triglyceride transfer protein inhibitor (lomitapide) efficacy in the treatment of patients with homozygous familial hypercholesterolaemia [published online August 12, 2019]. Eur J Prev Cardiol. doi: 10.1177/2047487319870007