Trial reportJournal of atherosclerosis and thrombosis2017
Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia.
Trial report in Journal of atherosclerosis and thrombosis, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Lomitapide, a Microsomal Triglyceride Transfer Protein Inhibitor, in Homozygous Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis of Efficacy and Safety.Cardiovascular drugs and therapy · 2026Pooled it
- Efficacy and Safety of Lomitapide in Homozygous Familial Hypercholesterolaemia: A Systematic Review.Reviews in cardiovascular medicine · 2022Pooled it
- Japan Atherosclerosis Society (JAS) Guidelines for Prevention of Atherosclerotic Cardiovascular Diseases 2017.Journal of atherosclerosis and thrombosis · 2018Guideline
- Safety and Efficacy of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH): Results from the AEGR-733-301 Long-Term Extension Study.Journal of atherosclerosis and thrombosis · 2019Trial
- Comparison of Clinical Characterization and Therapeutic Strategies between Homozygous Familial Hypercholesterolemia and Heterozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2026Article
- Liver Transplantation in a Child With Homozygous Familial Hypercholesterolemia: A Case Report and Literature Review.Reviews in cardiovascular medicine · 2025Review
- A New Insight on Atherosclerosis Mechanism and Lipid-Lowering Drugs.Reviews in cardiovascular medicine · 2025Review
- Japan Atherosclerosis Society (JAS) Guidelines for Prevention of Atherosclerotic Cardiovascular Diseases 2022.Journal of atherosclerosis and thrombosis · 2024Article
- Hypercholesterolemia: a literature review on management using tafolecimab: a novel member of PCSK9 monoclonal antibodies.Annals of medicine and surgery (2012) · 2024Review
- Lipoproteins, Cholesterol, and Atherosclerotic Cardiovascular Disease in East Asians and Europeans.Journal of atherosclerosis and thrombosis · 2023Review
- Guidelines for the Diagnosis and Treatment of Adult Familial Hypercholesterolemia 2022.Journal of atherosclerosis and thrombosis · 2023Article
- Gradual dose Titration of Lomitapide may Prevent Therapeutic Delays in Patients with Homozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2023Article
- Inflammation and atherosclerosis: signaling pathways and therapeutic intervention.Signal transduction and targeted therapy · 2022Review
- Individualized Treatment for Patients With Familial Hypercholesterolemia.Journal of lipid and atherosclerosis · 2022Review
- Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.Molecular therapy. Methods & clinical development · 2021Article
- Homozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2021Review
- Management of Familial Hypercholesterolemia: Current Status and Future Perspectives.Journal of the Endocrine Society · 2021Review
- Lomitapide-a Microsomal Triglyceride Transfer Protein Inhibitor for Homozygous Familial Hypercholesterolemia.Current atherosclerosis reports · 2020Review
- Lomitapide and Mipomersen-Inhibiting Microsomal Triglyceride Transfer Protein (MTP) and apoB100 Synthesis.Current atherosclerosis reports · 2019Review
- Familial Hypercholesterolemia and Lipoprotein Apheresis.Journal of atherosclerosis and thrombosis · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
aimThere 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.
methodsIn 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).
resultsNine 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.
conclusionLomitapide, 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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.