New England Journal of Medicine APRIL 03, 2008

Irbesartan in Heart Failure with Preserved Ejection Fraction (I-PRESERVE)

Massie BM, Carson PE, McMurray JJ, et al.

Bottom Line

The I-PRESERVE trial demonstrated that the angiotensin II receptor blocker irbesartan did not reduce the primary composite endpoint of all-cause mortality or cardiovascular hospitalization in patients with symptomatic heart failure and preserved ejection fraction (HFpEF) compared to placebo.

Key Findings

1. The primary composite endpoint of all-cause mortality or cardiovascular hospitalization occurred in 742 patients (36%) in the irbesartan group and 763 patients (37%) in the placebo group.
2. There was no statistically significant difference in the primary outcome, with a hazard ratio of 0.95 (95% CI, 0.86 to 1.05; P=0.35).
3. Individual components of the primary endpoint, including all-cause mortality and cardiovascular hospitalization, showed no significant benefit with irbesartan treatment.
4. The results were consistent across various prespecified subgroups, indicating no clear treatment benefit for irbesartan in the broader HFpEF population.

Study Design

Design
RCT
Double-Blind
Sample
4,128
Patients
Duration
49.5 mo
Median
Setting
25 countries
Population Patients aged 60 years or older with NYHA class II to IV symptoms, LVEF of 45% or higher, and either objective evidence of cardiac structural disease or a recent hospitalization for heart failure.
Intervention Irbesartan titrated to a target dose of 300 mg daily.
Comparator Matching placebo.
Outcome Composite of all-cause mortality or hospitalization for a predefined cardiovascular cause (worsening heart failure, unstable angina, myocardial infarction, ventricular or atrial dysrhythmia, or stroke).

Study Limitations

A significant proportion of patients (33%) discontinued the study drug, which may have impacted the ability to detect a smaller therapeutic effect.
High rates of concomitant use of other cardiovascular medications, including ACE inhibitors (40%), beta-blockers (73%), and spironolactone (39%), may have masked potential benefits of the intervention.
The trial was conducted in a population with diverse comorbidities and variable underlying causes of HFpEF, potentially diluting the effect of a single therapeutic approach.
The study design required symptomatic HF with preserved LVEF, but the inherent heterogeneity of HFpEF pathophysiology complicates the interpretation of negative results.

Clinical Significance

The study confirmed that neurohumoral blockade with an angiotensin receptor blocker, similar to findings with ACE inhibitors in previous HFpEF trials, does not improve clinical outcomes in patients with HFpEF, reinforcing the need for alternative therapeutic strategies.

Historical Context

At the time of the I-PRESERVE trial, the efficacy of renin-angiotensin-aldosterone system (RAAS) inhibitors in heart failure with reduced ejection fraction (HFrEF) was well-established. However, the management of heart failure with preserved ejection fraction remained an unmet challenge, with previous trials like CHARM-Preserved and PEP-CHF also failing to demonstrate clear survival or hospitalization benefits for RAAS inhibition in this specific patient population.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Given that RAAS inhibitors like irbesartan are cornerstone therapies for reducing mortality in heart failure with reduced ejection fraction (HFrEF), what pathophysiological differences in heart failure with preserved ejection fraction (HFpEF) might explain why irbesartan failed to show a similar benefit in the I-PRESERVE trial?

Key Response

While HFrEF is characterized by progressive ventricular dilation and neurohormonal-driven adverse remodeling, HFpEF is a more heterogeneous syndrome often driven by systemic inflammation, microvascular dysfunction, and stiffening of the myocardium due to comorbidities like obesity, hypertension, and diabetes. In HFpEF, the RAAS pathway may not be the primary driver of disease progression, making isolated RAAS blockade less effective than in the dilated, high-renin state of HFrEF.

Resident
Resident

Following the neutral results of the I-PRESERVE trial, how should a clinician approach the use of ARBs like irbesartan in a patient with symptomatic HFpEF and concomitant hypertension?

Key Response

The I-PRESERVE trial indicates that irbesartan should not be prescribed for the specific purpose of reducing cardiovascular mortality or heart failure hospitalizations in HFpEF. However, because hypertension is a major contributor to HFpEF pathogenesis and a common comorbidity, ARBs remain a first-line choice for blood pressure management in these patients. The goal in HFpEF remains symptom control (diuretics) and aggressive management of underlying risk factors rather than expecting a disease-modifying mortality benefit from the ARB itself.

Fellow
Fellow

The I-PRESERVE trial used an LVEF cutoff of ≥45%. How does the 'neutral' result of this trial compare to later findings in the 'mildly reduced' EF range (41-49%), and how might the inclusion of this subgroup have affected the study's overall outcome?

Key Response

Later evidence (such as the PARAGON-HF and TOPCAT subgroup analyses) suggests that patients at the lower end of the 'preserved' EF spectrum (specifically 40-55%) may derive some benefit from RAAS or neprilysin inhibition. By including a broad range of truly preserved EFs where diastolic dysfunction and non-cardiac comorbidities predominate, I-PRESERVE may have diluted potential signals of benefit that exist in the heart failure with mildly reduced ejection fraction (HFmrEF) phenotype.

Attending
Attending

How did the I-PRESERVE trial challenge the then-prevalent 'class effect' assumption that evidence-based therapies for HFrEF would naturally translate to benefit in HFpEF patients?

Key Response

I-PRESERVE was a landmark 'neutral' trial that provided a sobering lesson in evidence-based medicine: it demonstrated that HFpEF is a distinct clinical entity from HFrEF. It forced the cardiology community to move away from extrapolating HFrEF data and spurred the search for HFpEF-specific mechanisms, eventually leading to the study of SGLT2 inhibitors and ARNI, which finally showed efficacy where traditional RAAS blockade alone failed.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The I-PRESERVE trial observed a lower-than-predicted annual event rate in both the treatment and placebo arms. What are the statistical implications of this lower event rate on the study's power, and what enrichment strategies could modern researchers use to avoid this issue in HFpEF trials?

Key Response

A lower-than-expected event rate reduces the statistical power to detect a significant treatment effect, increasing the risk of a Type II error. To mitigate this in HFpEF research, modern trials utilize 'clinical enrichment'—selecting patients with elevated natriuretic peptides (NT-proBNP), recent HF hospitalizations, or objective structural markers (left atrial enlargement) to ensure the cohort has a high enough event rate to demonstrate therapeutic efficacy.

Journal Editor
Journal Editor

As a reviewer, how would you evaluate the impact of 'competing risks' from non-cardiovascular comorbidities on the primary composite endpoint of the I-PRESERVE trial?

Key Response

In older HFpEF populations, non-cardiovascular death is a significant 'competing risk' that can obscure the potential benefit of a cardiac intervention. A critical reviewer would flag that if a large proportion of the study population dies from non-cardiac causes (cancer, pulmonary disease, etc.), the study may be underpowered to show a difference in cardiovascular-specific outcomes, even if the drug has a biological effect on the heart.

Guideline Committee
Guideline Committee

How do the I-PRESERVE results reconcile with the current 2022 AHA/ACC/HFSA guideline recommendations for the use of ARBs in HFpEF patients?

Key Response

Current guidelines provide a Class 2b (Weak) recommendation for ARBs in HFpEF to potentially reduce hospitalizations, but this is primarily based on the CHARM-Preserved trial and TOPCAT (for spironolactone). I-PRESERVE reinforces the guideline stance that ARBs are not recommended for mortality reduction in this population (Level of Evidence: B-R). The guidelines prioritize SGLT2 inhibitors (Class 1) and suggest ARBs mainly for their role in managing hypertension (Class 1) rather than as a primary HFpEF treatment.

Clinical Landscape

Noteworthy Related Trials

2003

CHARM-Preserved Trial

n = 3,023 · Lancet

Tested

Candesartan

Population

Patients with symptomatic heart failure and LVEF >40%

Comparator

Placebo

Endpoint

Composite of CV death or hospital admission for worsening HF

Key result: Candesartan did not significantly reduce the primary composite endpoint compared to placebo, though it reduced hospitalizations for heart failure.
2014

TOPCAT Trial

n = 3,445 · NEJM

Tested

Spironolactone

Population

Patients with symptomatic heart failure and LVEF >45%

Comparator

Placebo

Endpoint

Composite of death from CV causes, aborted cardiac arrest, or hospitalization for heart failure

Key result: Spironolactone did not significantly reduce the primary composite endpoint in the overall population, although regional variations were observed.
2019

PARAGON-HF Trial

n = 4,822 · NEJM

Tested

Sacubitril/valsartan

Population

Patients with heart failure and LVEF >45%

Comparator

Valsartan

Endpoint

Total hospitalizations for heart failure and death from cardiovascular causes

Key result: Sacubitril/valsartan did not lead to a statistically significant reduction in the primary composite endpoint compared to valsartan alone.

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