Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (820 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
An azetidine derivative and anticholesteremic agent that inhibits intestinal sterol absorption. It is used to reduce total cholesterol; ldl cholesterol, and apolipoproteins B in the treatment of hyperlipidemias.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: (1-(4-fluorophenyl)-(3R)-(3-(4-fluorophenyl)-(3S)-hydroxypropyl)-(4S)-(4-hydroxyphenyl)-2-azetidinone); Ezetimib; SCH 58235; 58235, SCH; SCH-58235; SCH58235; Zetia; Ezetrol.
Where it fits in the MeSH classification
- Broader topics
- Azetidines
- Narrower topics
- Ezetimibe, Simvastatin Drug Combination
Related concepts
- Anticholesteremic Agent
- Sterols
- Cholesterol
- LDL Cholesterol
- Apolipoproteins
- Hyperlipidemias
Research landscape
PubMed indexes 2,965 records tagged with the MeSH heading “Ezetimibe” (counts retrieved 2026-10-31). 834 of them (28.1%) were published from 2020 onward, and the busiest period so far is 2010-2019 with 1,377 records.
Reviews account for 603 records (20.3%) and meta-analyses for 130. Randomized controlled trials number 586 (19.8%) as a share of all records on the topic. Randomized trials make up a comparatively large share of this literature, which means many treatment questions have been tested directly.
Show the data
| Period | Records |
|---|---|
| through 1999 | 0 |
| 2000-2009 | 788 |
| 2010-2019 | 1,377 |
| 2020 onward | 834 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 603 | 20.3% |
| Meta-analyses | 130 | 4.4% |
| Randomized controlled trials | 586 | 19.8% |
Method: PubMed query "Ezetimibe"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-31.
Reading the evidence in drugs and chemicals
Studies of a chemical or drug can answer very different questions: what it is and how it behaves in cells and animals, how the body handles it, and whether it changes outcomes in people. Laboratory and animal findings often do not carry over to patients, and observational studies of exposures are confounded by who uses a substance and why. Nothing on this page is a recommendation to use, avoid, start or stop any substance or medicine; talk to a qualified clinician or pharmacist about your own situation. Check the dose, the species, the comparison group and whether a patient-relevant outcome was measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
Ezetimibe Attenuates Oxidative Stress and Neuroinflammation via the AMPK/Nrf2/TXNIP Pathway after MCAO in Rats [1]
Abstract reading summary — source excerpts
- Opening excerpt
- Oxidative stress and neuroinflammation play essential roles in ischemic stroke-induced brain injury.
- Closing excerpt
- Therefore, Eze may be a potential therapeutic approach for ischemic stroke patients.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (195 words)
Oxidative stress and neuroinflammation play essential roles in ischemic stroke-induced brain injury. Previous studies have reported that Ezetimibe (Eze) exerts antioxidative stress and anti-inflammatory properties in hepatocytes. In the present study, we investigated the effects of Eze on oxidative stress and neuroinflammation in a rat middle cerebral artery occlusion (MCAO) model. One hundred and ninety-eight male Sprague-Dawley rats were used. Animals assigned to MCAO were given either Eze or its control. To explore the downstream signaling of Eze, the following interventions were given: AMPK inhibitor dorsomorphin and nuclear factor erythroid 2-related factor 2 (Nrf2) siRNA. Intranasal administration of Eze, 1 h post-MCAO, further increased the endogenous p-AMPK expression, reducing brain infarction, neurologic deficits, neutrophil infiltration, microglia/macrophage activation, number of dihydroethidium- (DHE-) positive cells, and malonaldehyde (MDA) levels. Specifically, treatment with Eze increased the expression of p-AMPK, Nrf2, and HO-1; Romo-1, thioredoxin-interacting protein (TXNIP), NOD-like receptor protein 3 (NLRP3), Cleaved Caspase-1, and IL-1β were reduced. Dorsomorphin and Nrf2 siRNA reversed the protective effects of Eze. In summary, Eze decreases oxidative stress and subsequent neuroinflammation via activation of the AMPK/Nrf2/TXNIP pathway after MCAO in rats. Therefore, Eze may be a potential therapeutic approach for ischemic stroke patients.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 31998437 · DOI · PMC6964721
Impact of ezetimibe on plasma lipoprotein(a) concentrations as monotherapy or in combination with statins: a systematic review and meta-analysis of randomized controlled trials [2]
Abstract reading summary — source excerpts
- Opening excerpt
- The aim of this meta-analysis of randomized placebo-controlled clinical trials was to assess the effect of ezetimibe on plasma lipoprotein(a) concentrations.
- Closing excerpt
- The results of this meta-analysis suggest that ezetimibe treatment either alone or in combination with a statin does not affect plasma lipoprotein(a) levels.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (203 words)
The aim of this meta-analysis of randomized placebo-controlled clinical trials was to assess the effect of ezetimibe on plasma lipoprotein(a) concentrations. Only randomized placebo-controlled trials investigating the impact of ezetimibe treatment on cholesterol lowering that include lipoprotein(a) measurement were searched in PubMed-Medline, SCOPUS, Web of Science and Google Scholar databases (from inception to February 26th, 2018). A random-effects model and generic inverse variance method were used for quantitative data synthesis. Sensitivity analysis was conducted using the leave-one-out method. A weighted random-effects meta-regression was performed to evaluate the impact of potential confounders on lipoprotein concentrations. This meta-analysis of data from 10 randomized placebo-controlled clinical trials (15 treatment arms) involving a total of 5188 (3020 ezetimibe and 2168 control) subjects showed that ezetimibe therapy had no effect on altering plasma Lp(a) concentrations (WMD: -2.59%, 95% CI: -8.26, 3.08, p = 0.370; I2 = 88.71%, p(Q) 2 = 65.38%, p(Q) = 0.005) or in trials evaluating the impact of adding ezetimibe to a statin versus statin therapy alone (WMD: -1.04%, 95% CI: -6.34, 4.26, p = 0.700; I2 = 58.51%, p(Q) = 0.025). The results of this meta-analysis suggest that ezetimibe treatment either alone or in combination with a statin does not affect plasma lipoprotein(a) levels.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 30552391 · DOI · PMC6294784
Non-statin lipid-lowering therapy over time in very-high-risk patients: effectiveness of fixed-dose statin/ezetimibe compared to separate pill combination on LDL-C [3]
Abstract reading summary — source excerpts
- Opening excerpt
- BackgroundMany patients at very-high atherosclerotic cardiovascular disease risk do not reach guideline-recommended targets for LDL-C.
- Closing excerpt
- The reduction in LDL-C when statin and ezetimibe were prescribed in combination was considerably larger for FDC; however, a large proportion of patients still remained with uncontrolled LDL-C levels.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (229 words)
BackgroundMany patients at very-high atherosclerotic cardiovascular disease risk do not reach guideline-recommended targets for LDL-C. There is a lack of data on real-world use of non-statin lipid-lowering therapies (LLT) and little is known on the effectiveness of fixed-dose combinations (FDC). We therefore studied prescription trends in oral non-statin LLT and their effects on LDL-C.MethodsA retrospective analysis was conducted of electronic medical records of outpatients at very-high cardiovascular risk treated by general practitioners (GPs) and cardiologists, and prescribed LLT in Germany between 2013 and 2018.ResultsData from 311,242 patients were analysed. Prescriptions for high-potency statins (atorvastatin and rosuvastatin) increased from 10.4% and 25.8% of patients treated by GPs and cardiologists, respectively, in 2013, to 34.7% and 58.3% in 2018. Prescription for non-statin LLT remained stable throughout the period and low especially for GPs. Ezetimibe was the most prescribed non-statin LLT in 2018 (GPs, 76.1%; cardiologists, 92.8%). Addition of ezetimibe in patients already prescribed a statin reduced LDL-C by an additional 23.8% (32.3 ± 38.4 mg/dL), with a greater reduction with FDC [reduction 28.4% (40.0 ± 39.1 mg/dL)] as compared to separate pills [19.4% (27.5 ± 33.8 mg/dL)]; p ConclusionsPrescription for high-potency statins increased over time. Non-statin LLT were infrequently prescribed by GPs. The reduction in LDL-C when statin and ezetimibe were prescribed in combination was considerably larger for FDC; however, a large proportion of patients still remained with uncontrolled LDL-C levels.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 32949286 · DOI · PMC8873069
The role of early ezetimibe combination with atorvastatin in patients with atherosclerotic cardiovascular disease [4]
Abstract reading summary — source excerpts
- Opening excerpt
- BackgroundDespite statin therapy, achieving target low-density lipoprotein cholesterol (LDL-C) levels remain suboptimal in high-risk patients with atherosclerotic cardiovascular disease (ASCVD).
- Closing excerpt
- Trial registrationNCT05761444; Registration date: March 9, 2023.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (193 words)
BackgroundDespite statin therapy, achieving target low-density lipoprotein cholesterol (LDL-C) levels remain suboptimal in high-risk patients with atherosclerotic cardiovascular disease (ASCVD). This study evaluated efficacy and safety of early addition of ezetimibe (EZ) with atorvastatin (AS), prior to reaching the maximally tolerated dose of statin, in very high-risk patients.MethodsThis phase 4 (NCT05761444), multicenter, randomized, open-label, active-controlled study enrolled patients (≥ 30 years) with very high-risk of ASCVD. Eligible patients had LDL-C ≥ 70 mg/dL with low/moderate intensity statin monotherapy or statin-naïve or not been on stable statin regimen prior to enrollment. Patients were randomized 1:1 to EZ10/AS40 mg combination therapy or AS40 mg statin alone for 12 weeks. Primary endpoint was percentage change in LDL-C from baseline to week 6.ResultsPatients (N = 137) received EZ/AS (n = 67) or AS (n = 70) once a day. The EZ/AS lipid-lowering effect was statistically greater than AS monotherapy at week 6 (LSMD: -21.2; P ConclusionsEarly combination of EZ with AS, rather than a stepwise approach, significantly reduced LDL-C levels and improved LDL-C reduction target achievement compared to AS monotherapy in very high-risk patients with dyslipidemia with no new safety issues.Trial registrationNCT05761444; Registration date: March 9, 2023.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41673582 · DOI · PMC12998120
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines [5]
- Research article 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol: Executive Summary: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines [6]
- Research article 2017 Focused Update of the 2016 ACC Expert Consensus Decision Pathway on the Role of Non-Statin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk: A Report of the American College of Cardiology Task Force on Expert Consensus Decision Pathways [7]
- Research article Efficacy and safety of lowering LDL cholesterol in older patients: a systematic review and meta-analysis of randomised controlled trials [8]
- Research article Effect of Ezetimibe Monotherapy on Plasma Lipoprotein(a) Concentrations in Patients with Primary Hypercholesterolemia: A Systematic Review and Meta-Analysis of Randomized Controlled Trials [9]
- Research article Optimal use of lipid-lowering therapy after acute coronary syndromes: A Position Paper endorsed by the International Lipid Expert Panel (ILEP) [10]
References
- Yu J, Wang WN, Matei N, Li X, Pang JW, Mo J, et al.. Ezetimibe Attenuates Oxidative Stress and Neuroinflammation via the AMPK/Nrf2/TXNIP Pathway after MCAO in Rats. . 2020. PMID 31998437 · DOI · PMC6964721
- Sahebkar A, Simental-Mendía LE, Pirro M, Banach M, Watts GF, Sirtori C, et al.. Impact of ezetimibe on plasma lipoprotein(a) concentrations as monotherapy or in combination with statins: a systematic review and meta-analysis of randomized controlled trials. . 2018. PMID 30552391 · DOI · PMC6294784
- Katzmann JL, Sorio-Vilela F, Dornstauder E, Fraas U, Smieszek T, Zappacosta S, et al.. Non-statin lipid-lowering therapy over time in very-high-risk patients: effectiveness of fixed-dose statin/ezetimibe compared to separate pill combination on LDL-C. . 2022. PMID 32949286 · DOI · PMC8873069
- Kang SH, Kwon SU, Lee JY, Seo SM, Nam CW, Park GM, et al.. The role of early ezetimibe combination with atorvastatin in patients with atherosclerotic cardiovascular disease. . 2026. PMID 41673582 · DOI · PMC12998120
- Grundy SM, Stone NJ, Bailey AL, Beam C, Birtcher KK, Blumenthal RS, et al.. 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines. J Am Coll Cardiol. 2019. PMID 30423393 · DOI
- Grundy SM, Stone NJ, Bailey AL, Beam C, Birtcher KK, Blumenthal RS, et al.. 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol: Executive Summary: A Report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines. J Am Coll Cardiol. 2019. PMID 30423391 · DOI
- Lloyd-Jones DM, Morris PB, Ballantyne CM, Birtcher KK, Daly DD, DePalma SM, et al.. 2017 Focused Update of the 2016 ACC Expert Consensus Decision Pathway on the Role of Non-Statin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk: A Report of the American College of Cardiology Task Force on Expert Consensus Decision Pathways. J Am Coll Cardiol. 2017. PMID 28886926 · DOI
- Gencer B, Marston NA, Im K, Cannon CP, Sever P, Keech A, et al.. Efficacy and safety of lowering LDL cholesterol in older patients: a systematic review and meta-analysis of randomised controlled trials. Lancet. 2020. PMID 33186535 · DOI · PMC8015314
- Awad K, Mikhailidis DP, Katsiki N, Muntner P, Banach M, Lipid and Blood Pressure Meta-Analysis Collaboration (LBPMC) Group. Effect of Ezetimibe Monotherapy on Plasma Lipoprotein(a) Concentrations in Patients with Primary Hypercholesterolemia: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Drugs. 2018. PMID 29396832 · DOI
- Banach M, Penson PE, Vrablik M, Bunc M, Dyrbus K, Fedacko J, et al.. Optimal use of lipid-lowering therapy after acute coronary syndromes: A Position Paper endorsed by the International Lipid Expert Panel (ILEP). Pharmacol Res. 2021. PMID 33607265 · DOI
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-31.
See how EvidenceLib is built. Spotted an error? Email support@evidencehelix.com.
Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.