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 W. Yancy, Peter E. Carson, Ralph B. D'Agostino Jr, et al.

Bottom Line

In self-identified Black patients with advanced heart failure, the addition of a fixed-dose combination of isosorbide dinitrate and hydralazine to standard neurohormonal blockade significantly improved a composite metric of survival, hospitalizations, and quality of life.

Key Findings

1. The trial was terminated early due to a significant mortality benefit; all-cause mortality was 6.2% in the ISDN-HYD group compared to 10.2% in the placebo group, representing a 43% relative reduction (Hazard Ratio, 0.57; P=0.01).
2. The mean primary composite score—incorporating mortality, hospitalization, and quality of life—was significantly higher in the intervention group (-0.1 ± 1.9) than in the placebo group (-0.5 ± 2.0) (P=0.01).
3. First hospitalization for heart failure was significantly reduced from 24.4% in the placebo group to 16.4% in the ISDN-HYD group (a 33% relative risk reduction; P=0.001).
4. Quality of life scores (Minnesota Living with Heart Failure questionnaire) significantly improved in the active treatment arm compared to placebo (-5.6 ± 20.6 vs. -2.7 ± 21.2; P=0.02).
5. Adverse events such as headache (47.5% vs. 19.2%) and dizziness (29.3% vs. 12.3%) were significantly more common with ISDN-HYD, 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 Self-identified Black patients with New York Heart Association (NYHA) class III or IV heart failure and dilated ventricles on optimal medical therapy.
Intervention Fixed-dose combination of isosorbide dinitrate and hydralazine (up to 120 mg ISDN and 225 mg HYD daily) added to standard therapy.
Comparator Placebo added to standard therapy.
Outcome A composite score weighting all-cause mortality, first hospitalization for heart failure, and change in quality of life at 6 months.

Study Limitations

The trial's early termination resulted in a median follow-up of only 10 months, which may exaggerate the perceived magnitude of the long-term mortality benefit.
The use of socially constructed, self-identified race as a proxy for genetic or pathophysiological traits (e.g., altered nitric oxide bioavailability) is imprecise and controversial.
Without a non-Black comparator arm in the same prospective trial, it remains unclear whether the observed benefits of ISDN-HYD are biologically unique to this demographic or if the trial simply unmasked an under-recognized benefit applicable to broader populations.
High rates of vasodilatory adverse events, particularly headache and dizziness, present significant challenges for patient tolerability and long-term adherence in real-world clinical practice.

Clinical Significance

A-HeFT established fixed-dose isosorbide dinitrate and hydralazine as a Class I guideline recommendation for self-identified Black patients with NYHA class III-IV heart failure with reduced ejection fraction who remain symptomatic despite optimal medical therapy. The trial successfully demonstrated that addressing distinct pathophysiological mechanisms—specifically nitric oxide depletion and increased oxidative stress—can yield substantial morbidity and mortality benefits.

Historical Context

Prior subgroup analyses from the V-HeFT I and II trials in the 1980s and 1990s suggested that Black patients might derive a disproportionate benefit from vasodilator therapy with hydralazine and isosorbide dinitrate, particularly since they frequently exhibited less robust clinical responses to standard ACE inhibitors. The A-HeFT trial was explicitly designed to test this hypothesis prospectively. Following its publication, the FDA approved the fixed-dose combination (BiDil) specifically for self-identified Black patients, marking a contentious milestone as the first race-specific drug indication in modern medicine and sparking intense debate over pharmacogenomics and the use of race in medical research.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the pathophysiologic rationale for combining hydralazine with isosorbide dinitrate, particularly regarding endothelial function and oxidative stress?

Key Response

Isosorbide dinitrate acts as a nitric oxide (NO) donor, promoting venodilation and reducing preload. Hydralazine acts as an arterial vasodilator to reduce afterload, but crucially, it also functions as an antioxidant. By reducing oxidative stress, hydralazine prevents the rapid degradation of NO, creating a synergistic effect that is especially beneficial in pathophysiological states where endogenous NO bioavailability is impaired.

Resident
Resident

In clinical practice, what are the most common adverse effects of the hydralazine/isosorbide dinitrate combination that limit patient adherence, and how should a clinician manage them?

Key Response

The combination frequently causes headaches, dizziness, and gastrointestinal distress, leading to high discontinuation rates. Clinicians must actively manage this by starting at lower doses, titrating slowly, and educating patients that the headaches are often transient and can be managed with over-the-counter analgesics initially to maintain adherence to this mortality-reducing therapy.

Fellow
Fellow

Given the success of A-HeFT in Black patients, how should a heart failure specialist approach the use of hydralazine/isosorbide dinitrate in non-Black patients with HFrEF?

Key Response

While A-HeFT focused on self-identified Black patients due to retrospective signals from V-HeFT I/II, hydralazine/ISDN is frequently used off-label in non-Black patients who are intolerant to ACEi/ARB/ARNI (e.g., due to severe renal dysfunction or hyperkalemia) or who remain symptomatic despite guideline-directed medical therapy. Fellows must weigh the lack of targeted prospective trial data in this demographic against the physiologic rationale and need for salvage therapy.

Attending
Attending

The A-HeFT trial introduced the concept of race-based therapeutics in cardiology. As an attending, how do you contextualize and teach the limitations of using a social construct like self-identified race as a proxy for pharmacogenomics?

Key Response

Race is a social, not biological, construct. Using it as a proxy for reduced nitric oxide bioavailability or specific genetic variants is imprecise. Attendings must teach trainees to recognize the historical significance of A-HeFTs mortality benefit while advocating for a transition toward precision medicine that targets actual physiological markers or genetic profiles rather than relying on broad racial categories.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The A-HeFT trial was terminated early due to a significant mortality benefit. What are the statistical and epidemiological risks of early trial termination for benefit, and how might it affect the estimation of the true treatment effect size?

Key Response

Early termination for benefit often leads to an overestimation of the treatment effect, a phenomenon known as the random high or regression to the mean. Researchers must critically evaluate whether the stopping rules (e.g., OBrien-Fleming boundaries) were appropriately conservative, and consider how early stopping limits the statistical power of secondary endpoints and the collection of long-term safety data.

Journal Editor
Journal Editor

If reviewing the A-HeFT manuscript today, how would you critically appraise the use of a composite primary endpoint that combines mortality, first heart failure hospitalization, and change in quality of life score?

Key Response

Editors must scrutinize composite endpoints to ensure the components have similar clinical importance and directionality of effect. Mixing a hard objective endpoint like mortality with a softer subjective one like a quality of life score can obscure true effects or drive a positive trial purely through the softer metric. Reviewers must demand a clear breakdown of each component to ensure there is no masking or endpoint creep.

Guideline Committee
Guideline Committee

Based on the A-HeFT trial, what is the current ACC/AHA/HFSA guideline recommendation for the use of hydralazine and isosorbide dinitrate in self-identified African American patients, and how does modern background therapy complicate this?

Key Response

Current guidelines give a Class 1 recommendation for adding hydralazine and ISDN to self-identified African American patients with NYHA class III-IV HFrEF receiving optimal medical therapy. The committee must consider that optimal therapy during A-HeFT did not include ARNIs or SGLT2 inhibitors. Updating guidelines requires evaluating whether the absolute benefit of this combination persists on top of modern, more robust quadruple therapy.

Clinical Landscape

Noteworthy Related Trials

1986

V-HeFT I

n = 642 · NEJM

Tested

Hydralazine plus isosorbide dinitrate

Population

Men with impaired left ventricular function and heart failure

Comparator

Placebo or prazosin

Endpoint

All-cause mortality

Key result: The combination of hydralazine and isosorbide dinitrate reduced mortality at 2 years compared to placebo, whereas prazosin had no effect.
1991

V-HeFT II

n = 804 · NEJM

Tested

Enalapril

Population

Men with chronic heart failure taking digoxin and diuretics

Comparator

Hydralazine plus isosorbide dinitrate

Endpoint

All-cause mortality

Key result: Enalapril significantly reduced 2-year mortality compared to the hydralazine and isosorbide dinitrate combination, establishing ACE inhibitors as superior first-line therapy.
1991

SOLVD-Treatment Trial

n = 2,569 · NEJM

Tested

Enalapril

Population

Patients with symptomatic heart failure and an ejection fraction of 35 percent or less

Comparator

Placebo

Endpoint

All-cause mortality

Key result: The addition of enalapril to conventional therapy significantly reduced mortality and hospitalizations for heart failure.

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