Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia
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- Harada-Shiba Mariko
- National Cerebral and Cardiovascular Research Center
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- Ikewaki Katsunori
- National Defense Medical College
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- Nohara Atsushi
- Kanazawa University Graduate School of Medical Sciences
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- Otsubo Yoshihiko
- Shin-Koga Clinic, Medical Group Tenjin-kai
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- Yanagi Koji
- Kenporen Osaka Central Hospital
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- Yoshida Masayuki
- Tokyo Medical and Dental University
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- Chang Qing
- Aegerion Pharmaceuticals, Inc.
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- Foulds Pamela
- Aegerion Pharmaceuticals, Inc.
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Abstract
<p>Aim: There is an unmet need in Japan for more optimal lipid-lowering therapy (LLT) for patients with homozygous familial hypercholesterolemia (HoFH) who respond inadequately to available drug therapies and/or apheresis, to achieve goals of low-density lipoprotein cholesterol (LDL-C) reduction by 50% or to ＜100 mg/dL.</p><p>Methods: In this study, Japanese patients with HoFH on stable LLT and diet were treated with lomitapide, initiated at 5 mg/day and escalated to maximum tolerated dose (up to 60 mg/day) over 14 weeks. The primary efficacy endpoint was mean percentage change from baseline to Week 26 in LDL-C. Secondary endpoints included changes in other lipid parameters and safety throughout the 56-week study (including follow-up).</p><p>Results: Nine patients entered the efficacy phase of the study and, of these, eight completed 56 weeks. Mean LDL-C was reduced by 42% (p＜0.0001) at 26 weeks, from 199 mg/dL (95% CI: 149–250) at baseline to 118 mg/dL (95% CI: 70–166). A 50% reduction in LDL-C and LDL-C ＜100 mg/dL was achieved by five and six of nine patients, respectively, at 26 weeks. After 56 weeks, LDL-C was reduced by 38% (p=0.0032) from baseline. Significant reductions in non-HDL-C, VLDL-C, triglycerides, and apolipoprotein B were also reported at Week 26. There were no new safety signals and, similar to previous studies, gastrointestinal adverse events were the most common adverse events.</p><p>Conclusion: Lomitapide, added to ongoing treatment with other LLTs, was effective in rapidly and significantly reducing the levels of LDL-C and other atherogenic apolipoprotein B-containing lipoproteins in adult Japanese patients with HoFH.</p>
Journal
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- Journal of Atherosclerosis and Thrombosis
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Journal of Atherosclerosis and Thrombosis 24 (4), 402-411, 2017
Japan Atherosclerosis Society
