Evidence map›Paper›PMID 40856940›Full record

SynthesisCardiovascular drugs and therapy2026

Lomitapide, a Microsomal Triglyceride Transfer Protein Inhibitor, in Homozygous Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis of Efficacy and Safety.

Mohamed Nasser, Hazem E Mohammed, Mohamed E Haseeb, Mohamed Khalafalla Darwish, George Hanen, Nada A Abdelaziz, Anas Hussien Heiba, Abdelrahman Shata, Mohamed Salem Abdelkader

Abstract readSystematic ReviewMeta-Analysis
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Cardiovascular drugs and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

The trial behind it

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Mohamed Nasser *Faculty of Medicine, Minia University, Minia, Egypt. MohamedNasser10117@gmail.com.ORCID 0009-0000-9111-0392
Hazem E Mohammed *Faculty of Medicine, Assiut University, Assiut, Egypt.
Mohamed E HaseebFaculty of Medicine, Minia University, Minia, Egypt.
Mohamed Khalafalla DarwishFaculty of Medicine, Minia University, Minia, Egypt.
George HanenFaculty of Medicine, Minia University, Minia, Egypt.
Nada A AbdelazizFaculty of Medicine, Minia University, Minia, Egypt.
Anas Hussien HeibaFaculty of Medicine, Minia University, Minia, Egypt.
Abdelrahman ShataFaculty of Medicine, Horus University-Egypt, New Damietta, Egypt.
Mohamed Salem AbdelkaderFaculty of Medicine, Minia University, Minia, Egypt.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeHomozygous Familial Hypercholesterolemia (HoFH) is a rare and life-threatening genetic disorder characterized by elevated low-density lipoprotein cholesterol (LDL-C) and early-onset atherosclerotic cardiovascular disease. Lomitapide, a microsomal triglyceride transfer protein (MTP) inhibitor, decreases LDL-C independent of LDL receptor function, providing an alternative treatment in this population. We aimed to evaluate the efficacy and safety of lomitapide in patients with HoFH through a systematic review and meta-analysis of available clinical evidence.

methodsA comprehensive search was conducted in PubMed, Scopus, and Web of Science through March 2025. Observational studies and clinical trials reporting on lipid profile changes and safety outcomes in HoFH patients receiving lomitapide were included. Outcomes were pooled using random-effects models, and heterogeneity was assessed using the I

resultsEight studies comprising both adult and pediatric patients (n = 209) were included. Lomitapide significantly reduced LDL-C levels by 49.27%, total cholesterol by 46.05%, and apolipoprotein B by 51.01%. Reductions were also observed in triglycerides, VLDL-C, and non-HDL-C. HDL-C remained relatively unchanged. Adverse events were mostly gastrointestinal, with a 14% discontinuation rate. The overall quality of studies ranged from fair to good.

conclusionsLomitapide demonstrates substantial efficacy in reducing LDL-C and other atherogenic lipids in HoFH patients, with an acceptable safety profile. These findings support its role as an adjunctive therapy in this population, though further randomized controlled trials are warranted to validate long-term safety and effectiveness.

Indexed as

Anticholesteremic AgentsBenzimidazolesCarrier ProteinsCholesterol, LDLHyperlipoproteinemia Type IIBiomarkersHomozygoteHumansTreatment OutcomeAnticholesteremic AgentsBenzimidazolesBiomarkersBMS201038Carrier ProteinsCholesterol, LDLmicrosomal triglyceride transfer proteinHoFHHomozygous Familial HypercholesterolemiaLomitapideMeta-analysisMicrosomal triglyceride transfer protein inhibitorSystematic review

Identifiers

PMID40856940
PMCPMC13171640

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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.