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Ez-PAVE

Effects of Ezetimibe Combination Therapy for Patients with Atherosclerotic Cardiovascular Disease — Randomized Comparison of LDL Cholesterol Targeting <70 mg per Deciliter vs. <55 mg per Deciliter

Year of Publication: 2026

Authors: Yong-Joon Lee, Seung-Jun Lee, Jin Won Kim, ..., Byeong-Keuk Kim

Journal: New England Journal of Medicine

Citation: N Engl J Med 2026;394:1365-75

Link: https://doi.org/10.1056/NEJMoa2600283


Clinical Question

Among patients with atherosclerotic cardiovascular disease, is targeting an LDL cholesterol level of less than 55 mg per deciliter superior to targeting a level of less than 70 mg per deciliter for preventing recurrent major cardiovascular events?

Bottom Line

In patients with atherosclerotic cardiovascular disease, targeting an LDL cholesterol level of less than 55 mg/dL resulted in a 33% lower risk of cardiovascular events at 3 years compared to targeting less than 70 mg/dL (HR 0.67, 95% CI 0.52-0.86, p=0.002), with similar safety profiles except for lower creatinine elevation in the intensive-targeting group.

Major Points

  • Primary composite endpoint (cardiovascular death, nonfatal MI, nonfatal stroke, any revascularization, or hospitalization for unstable angina) occurred in 6.6% of intensive-targeting group vs. 9.7% of conventional-targeting group at 3 years (HR 0.67, 95% CI 0.52-0.86, p=0.002)
  • Median LDL cholesterol achieved during trial was 56 mg/dL in intensive-targeting group vs. 66 mg/dL in conventional-targeting group
  • Nonfatal MI occurred in 0.8% vs. 1.7% (HR 0.46, 95% CI 0.23-0.91)
  • Any revascularization occurred in 4.8% vs. 7.5% (HR 0.63, 95% CI 0.47-0.84)
  • At 3 years, 60.8% of intensive-targeting group achieved LDL <55 mg/dL, and 85.2% achieved <70 mg/dL
  • At 3 years, 48.4% of intensive-targeting group received high-intensity statins, 66.6% received ezetimibe, and 2.3% received PCSK9 inhibitors
  • No significant differences in safety outcomes except lower creatinine elevation in intensive-targeting group (1.2% vs. 2.7%, p=0.004)
  • Similar incidence of new-onset diabetes, muscle symptoms, cancer diagnosis, and liver enzyme elevation between groups

Design

Study Type: Randomized controlled trial

Randomization: 1

Blinding: Open-label

Allocation: 1:1 randomization stratified by previous acute coronary syndrome, presence of diabetes, and baseline LDL cholesterol level, using permuted-block randomization with mixed blocks of 4 or 6

Enrollment Period: January 2021 through July 2022

Follow-up Duration: Median 3.0 years (IQR 3.0-3.0)

Centers: 17

Countries: South Korea

Sample Size: 3048

Analyzed: 3048

Analysis: Intention-to-treat analysis with per-protocol sensitivity analysis

Power Calculation: Sample size of 3048 patients provided 80% power at two-sided alpha of 0.05 to detect 24.75% lower relative risk of primary endpoint at 3 years, assuming 15% incidence in conventional-targeting group and 15% loss to follow-up

Registration: NCT04626973


Inclusion Criteria

  • Age 19 to 80 years
  • Documented atherosclerotic cardiovascular disease defined as at least one of:
  • Previous acute coronary syndrome (myocardial infarction or unstable angina)
  • Stable angina with imaging or functional studies
  • Coronary revascularization or other arterial revascularization
  • Stroke or transient ischemic attack
  • Peripheral artery disease

Exclusion Criteria

  • LDL cholesterol level less than 70 mg/dL without statin therapy (key exclusion criterion)
  • Additional exclusion criteria detailed in Supplementary Appendix

Arms

FieldControlIntensive Targeting
N15221526
InterventionTarget LDL cholesterol level of less than 70 mg per deciliter using statins, ezetimibe, and PCSK9 inhibitors as neededTarget LDL cholesterol level of less than 55 mg per deciliter using statins, ezetimibe, and PCSK9 inhibitors as needed
Duration3 years3 years

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Composite of death from cardiovascular causes, nonfatal myocardial infarction, nonfatal stroke, any revascularization, or hospitalization for unstable anginaPrimary147 patients (9.7% cumulative incidence)100 patients (6.6% cumulative incidence)0.670.002
Death from cardiovascular causesSecondary18 patients (1.2%)15 patients (1.0%)Not specified
Nonfatal myocardial infarctionSecondary26 patients (1.7%)12 patients (0.8%)Not specified
Nonfatal strokeSecondary10 patients (0.7%)8 patients (0.5%)Not specified
Ischemic strokeSecondary7 patients (0.5%)7 patients (0.5%)Not specified
Hemorrhagic strokeSecondary3 patients (0.2%)1 patient (0.1%)Not specified
Any revascularizationSecondary113 patients (7.5%)72 patients (4.8%)Not specified
Percutaneous coronary interventionSecondary99 patients (6.6%)67 patients (4.5%)Not specified
Coronary-artery bypass graftingSecondary7 patients (0.5%)1 patient (0.1%)Not specified
Other arterial revascularizationSecondary9 patients (0.6%)6 patients (0.4%)Not specified
Hospitalization for unstable anginaSecondary36 patients (2.4%)22 patients (1.5%)Not specified
Composite of cardiovascular death, nonfatal MI, or nonfatal strokeSecondary54 patients (3.6%)34 patients (2.3%)Not specified
Composite of cardiovascular death, nonfatal MI, nonfatal stroke, or any revascularizationSecondary141 patients (9.3%)95 patients (6.3%)Not specified
Composite of cardiovascular death, nonfatal MI, or any revascularizationSecondary132 patients (8.7%)88 patients (5.8%)Not specified
Composite of all-cause death, nonfatal MI, nonfatal stroke, any revascularization, or hospitalization for unstable anginaSecondary157 patients (10.4%)116 patients (7.6%)Not specified
Death from any causeSecondary29 patients (1.9%)31 patients (2.0%)Not specified
New-onset diabetes (among patients without diabetes at baseline)Safety148/920 (16.1%)153/921 (16.6%)0.81
Worsening of glycemic control (among patients with diabetes at baseline)Safety305/602 (50.7%)295/605 (48.8%)0.55
Statin-associated muscle symptoms leading to therapy changesSafety9 patients (0.6%)15 patients (1.0%)0.31
Diagnosis of cancerSafety40 patients (2.6%)36 patients (2.4%)0.72
Cataract surgerySafety16 patients (1.1%)20 patients (1.3%)0.62
Aminotransferase elevation (>3× ULN)Safety23 patients (1.5%)37 patients (2.4%)0.09
Creatinine elevation (>1.5× baseline)Safety41/1515 (2.7%)18/1517 (1.2%)0.004
Creatine kinase elevation (>4× ULN)Safety4 patients (0.3%)4 patients (0.3%)1.00
Discontinuation of LDL cholesterol-lowering therapy due to adverse eventsAdverse35 patients (2.3%)50 patients (3.3%)
Discontinuation or downward adjustment despite not reaching targetAdverse48 patients (3.2%)62 patients (4.1%)

Subgroup Analysis

Subgroup analyses showed consistent benefit of intensive targeting across most prespecified subgroups including age, sex, BMI, previous ACS, revascularization history, stroke/TIA, peripheral artery disease, hypertension, diabetes, chronic kidney disease, and baseline LDL cholesterol level. No significant interactions were detected.


Criticisms

  • Open-label design may introduce bias, though endpoint adjudication was blinded
  • Actual number of primary endpoint events was lower than anticipated in sample size calculation
  • Only 60.8% of intensive-targeting group reached target LDL <55 mg/dL at 3 years
  • Limited use of PCSK9 inhibitors (only 2.3% at 3 years in intensive-targeting group) due to reimbursement constraints
  • Other nonstatin agents (inclisiran, bempedoic acid) were unavailable during trial period
  • LDL cholesterol levels analyzed without imputation for missing data, potentially introducing bias
  • 3-year follow-up may be insufficient to evaluate long-term effects
  • Trial conducted only in East Asian patients, limiting generalizability to other populations
  • 39% of intensive-targeting group did not reach target at 3 years, suggesting real-world implementation challenges

Funding

Funded by the Cardiovascular Research Center (South Korea) and Yuhan. The funders had no role in trial design, data collection, analysis, interpretation, or manuscript preparation.

Based on: Ez-PAVE (New England Journal of Medicine, 2026)

Authors: Yong-Joon Lee, Seung-Jun Lee, Jin Won Kim, ..., Byeong-Keuk Kim

Citation: N Engl J Med 2026;394:1365-75

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