The New England Journal of Medicine NOVEMBER 11, 2004

Combination of Isosorbide Dinitrate and Hydralazine in Blacks with Heart Failure

Anne L. Taylor, Susan Ziesche, Clyde Yancy, Peter Carson, Francisco D'Agostino, Krishnaswamy Krishnan, Michael Ellis, Martha Gonze, Jay N. Cohn

Bottom Line

In African American patients with advanced heart failure receiving standard therapy, the addition of a fixed-dose combination of isosorbide dinitrate and hydralazine significantly improved survival, reduced the risk of first hospitalization for heart failure, and enhanced quality of life.

Key Findings

1. The trial was terminated early for efficacy after a clear mortality benefit was observed in the intervention group compared with the placebo group (6.2% vs. 10.2%; hazard ratio 0.57; P=0.01).
2. Patients receiving the combination therapy experienced a significantly lower rate of first hospitalization for heart failure compared to those in the placebo group (16.4% vs. 24.4%; P=0.001).
3. The study met its primary composite endpoint, demonstrating a significant improvement in clinical outcomes including mortality, hospitalizations, and quality of life scores (P=0.01).
4. Adverse events such as headache (47.5% vs. 19.2%; P<0.001) and dizziness (29.3% vs. 12.3%; P<0.001) were significantly more frequent in the treatment group, though exacerbations of heart failure were lower.

Study Design

Design
RCT
Double-Blind
Sample
1,050
Patients
Duration
10 mo
Median
Setting
Multicenter, US
Population Patients identifying as African American with NYHA class III or IV heart failure receiving standard therapy for heart failure.
Intervention Fixed-dose combination of isosorbide dinitrate (20 mg) and hydralazine (37.5 mg) taken three times daily, titrated to a maximum of 120 mg/225 mg daily.
Comparator Placebo added to standard background heart failure therapy (including ACE inhibitors/ARBs, beta-blockers, and diuretics).
Outcome A composite score incorporating all-cause mortality, first hospitalization for heart failure, and change in quality of life scores.

Study Limitations

The trial was limited to patients who self-identified as African American, which restricts the direct generalizability of the findings to other racial or ethnic groups.
The study employed a novel composite score as the primary endpoint, which, while capturing broad clinical impact, complicates the interpretation of individual component contributions.
The trial was stopped early by the data and safety monitoring board, which can sometimes lead to an overestimation of the treatment effect size in clinical trials.
Although standard background therapy was high, the study was conducted during an era where current contemporary therapies like SGLT2 inhibitors and ARNI were not part of the standard regimen.

Clinical Significance

This landmark study established a race-specific therapeutic option for advanced heart failure, becoming the first clinical trial to demonstrate clear benefit of a fixed-dose vasodilator combination (BiDil) added to standard therapy in African American patients, leading to its inclusion in major heart failure clinical guidelines.

Historical Context

The A-HeFT trial was designed to prospectively test observations from retrospective analyses of the earlier V-HeFT trials, which suggested that African American patients derived a greater survival benefit from hydralazine and isosorbide dinitrate than white patients, a finding attributed to potential differences in nitric oxide bioavailability and renin-angiotensin system activity.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Based on the mechanisms of action, why is the combination of hydralazine and isosorbide dinitrate particularly effective in treating the pathophysiology of heart failure?

Key Response

Hydralazine acts as an arterial vasodilator (reducing afterload) and an antioxidant that prevents the degradation of nitric oxide. Isosorbide dinitrate is a venous vasodilator (reducing preload) that acts as a nitric oxide donor. Together, they address the deficiency in nitric oxide bioavailability and the high oxidative stress often observed in the heart failure phenotype studied in A-HeFT.

Resident
Resident

Which specific patient population from the A-HeFT trial should be considered for the addition of fixed-dose isosorbide dinitrate and hydralazine, and what was the magnitude of the mortality benefit observed?

Key Response

The trial specifically enrolled self-identified Black patients with NYHA Class III or IV heart failure with reduced ejection fraction (HFrEF) who were already on standard therapy (ACE inhibitors, beta-blockers). The study demonstrated a striking 43% relative reduction in the rate of death from any cause, necessitating a low number needed to treat (NNT) to prevent one death.

Fellow
Fellow

Contrast the findings of A-HeFT with the earlier V-HeFT I and II trials. How did the patient selection in A-HeFT address the limitations or subgroups identified in those prior studies?

Key Response

V-HeFT I showed hydralazine/ISDN was better than placebo, but V-HeFT II showed it was inferior to enalapril in a general population. However, post-hoc subgroup analyses of V-HeFT suggested that Black patients had a more robust response to the combination than white patients. A-HeFT was specifically designed to prospectively validate this observation, focusing on a population where nitric oxide signaling may be more significantly impaired.

Attending
Attending

How does the A-HeFT trial serve as a landmark for 'precision medicine' or 'pharmacogenetics' in cardiology, and what are the ethical implications of race-based prescribing in modern practice?

Key Response

A-HeFT led to the first race-specific FDA drug approval (BiDil). While it demonstrates the power of tailoring therapy to populations with specific biological responses, it also prompts a debate on whether race is a biological reality or a social proxy for unmeasured genetic or environmental factors, highlighting the need for future trials to move toward genomic or biomarker-driven inclusion criteria.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

Critique the use of 'self-identified race' as the primary inclusion criterion in A-HeFT from a genetic epidemiology perspective. What are the risks of using such a proxy compared to measuring specific eNOS polymorphisms or markers of oxidative stress?

Key Response

Self-identified race is a multidimensional construct that incorporates social, environmental, and genetic factors. Using it as a proxy for drug response assumes a genetic homogeneity that may not exist. A more rigorous approach would involve genotyping for variants in the endothelial nitric oxide synthase (eNOS) gene or measuring urinary/plasma markers of NO metabolism to define the 'responder' phenotype more accurately and avoid confounding by social determinants of health.

Journal Editor
Journal Editor

The A-HeFT trial was terminated early by the data and safety monitoring board. As a reviewer, how would you evaluate the risk of 'overestimation of effect' (the winner's curse) and its impact on the reported secondary endpoints like quality-of-life scores?

Key Response

Trials stopped early for benefit are notorious for inflating the treatment effect size. While the primary endpoint (a composite score) and mortality were statistically robust, the secondary endpoints—particularly subjective measures like the Minnesota Living with Heart Failure questionnaire—may show an exaggerated benefit because the trial ended before regression to the mean could occur in the treatment arm.

Guideline Committee
Guideline Committee

Compare the ACC/AHA Class of Recommendation for isosorbide dinitrate/hydralazine in Black patients versus its recommendation for the general population with HFrEF who are intolerant to ACE/ARBs.

Key Response

Current guidelines (ACC/AHA/HFSA) provide a Class 1 (Level A) recommendation for adding ISDN/hydralazine to Black patients with NYHA Class III-IV HFrEF on GDMT based on A-HeFT. In contrast, for the general population, it is a Class 2b recommendation for patients who cannot tolerate ACE inhibitors or ARBs (due to renal failure or hyperkalemia), reflecting the weaker evidence base from the older V-HeFT trials for non-Black populations.

Clinical Landscape

Noteworthy Related Trials

1986

V-HeFT I Trial

n = 642 · NEJM

Tested

Hydralazine plus isosorbide dinitrate

Population

Men with symptomatic heart failure

Comparator

Placebo or Prazosin

Endpoint

Mortality

Key result: The combination of hydralazine and isosorbide dinitrate significantly reduced mortality compared to placebo.
1991

V-HeFT II Trial

n = 804 · NEJM

Tested

Enalapril

Population

Men with symptomatic heart failure

Comparator

Hydralazine plus isosorbide dinitrate

Endpoint

Mortality

Key result: Enalapril was found to be superior to the combination of hydralazine and isosorbide dinitrate in reducing mortality.
1991

SOLVD-Treatment Trial

n = 2,569 · NEJM

Tested

Enalapril

Population

Patients with left ventricular dysfunction and heart failure

Comparator

Placebo

Endpoint

All-cause mortality

Key result: Enalapril significantly reduced the risk of death and the rate of hospitalization for heart failure.

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