Marina Cuchel, Dirk J. Blom, Maurizio R. Averna

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Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia Marina Cuchel, Dirk J. Blom, Maurizio R. Averna Atherosclerosis Supplements Volume 15, Issue 2, Pages 33-45 (September 2014) DOI: 10.1016/j.atherosclerosissup.2014.07.005 Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 1 Lomitapide inhibits the microsomal triglyceride transfer protein (MTP) activity in the liver and in the intestine. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 2 Design of phase 3 study of lomitapide in patients with homozygous familial hypercholesterolaemia. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 3 Low-density lipoprotein (LDL) cholesterol levels of patients at baseline. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 4 Percentage reduction in concentration of low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (apoB) and total cholesterol (TC) levels with lomitapide in addition to maximum lipid-lowering therapy (A); individual patient LDL-C responses by dose of lomitapide (B). A. Data are mean, 95%CI (n = 23). B. #Patient was a responder at later time points during the study. ‡Patients discontinued from the study. Numbers over the bars are baseline LDL-C in mmol/L. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 5 Case study 1: Low-density lipoprotein cholesterol (LDL-C) level in a 44 year old woman with homozygous familial hypercholesterolaemia receiving lomitapide in phase 3 study. The dose of lomitapide was uptitrated to 60 mg during the efficacy phase of the study (Weeks 0–26). Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 6 Case study 2. Low-density lipoprotein cholesterol (LDL-C) and total cholesterol levels in a patient receiving lomitapide in phase 3 study. From lomitapide efficacy phase (A), through to extension phase (B). Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 7 Case study 3. Baseline levels of transaminases (ALT and AST) before treatment with lomitapide. The run-in period for this patient was longer than typical due to an AST/ALT flare associated with the first administration of lomitapide. The drug was stopped and successfully reintroduced 9 weeks later. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 8 Case study #3: Concentrations of transaminases (ALT and AST) during efficacy phase (A), safety phase (B) and transaminitis flare (C). Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 9 Case study 3: Concentrations of transaminases (ALT and AST) during rechallenge with lomitapide. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions

Fig. 10 Case study 4. Mean low-density lipoprotein (LDL) cholesterol levels in a patient receiving treatment with lomitapide, with or without LDL apheresis, in the phase 3 study. Atherosclerosis Supplements 2014 15, 33-45DOI: (10.1016/j.atherosclerosissup.2014.07.005) Copyright © 2014 Elsevier Ireland Ltd Terms and Conditions