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, Ralph D'Agostino, et al.

Bottom Line

The addition of a fixed-dose combination of isosorbide dinitrate and hydralazine to standard medical therapy in Black patients with advanced heart failure significantly improved a primary composite score, enhanced quality of life, and reduced both all-cause mortality and heart failure hospitalizations.

Key Findings

1. The trial was terminated early due to a significantly lower mortality rate in the isosorbide dinitrate plus hydralazine group compared to the placebo group (6.2% vs. 10.2%, P=0.02).
2. The mean primary composite score (which weighted death, heart failure hospitalization, and quality of life) was significantly better in the intervention group (-0.1 ± 1.9) than in the placebo group (-0.5 ± 2.0) (P=0.01).
3. Treatment resulted in a 43% reduction in the rate of death from any cause (hazard ratio, 0.57; P=0.01).
4. There was a 33% relative reduction in the rate of a first hospitalization for heart failure in the intervention arm (16.4% vs. 22.4%, P=0.001).
5. Quality of life scores significantly improved in the intervention group compared to placebo (change in score, -5.6 ± 20.6 vs. -2.7 ± 21.2, P=0.02, with lower scores indicating better quality of life).

Study Design

Design
RCT
Double-Blind
Sample
1,050
Patients
Duration
10 mo
Median
Setting
Multicenter, US
Population Black patients with New York Heart Association class III or IV heart failure and dilated ventricles
Intervention Fixed dose of isosorbide dinitrate plus hydralazine in addition to standard heart failure therapy
Comparator Placebo in addition to standard heart failure therapy
Outcome A composite score consisting of weighted values for death from any cause, a first hospitalization for heart failure, and change in quality of life

Study Limitations

• The trial was terminated early (mean follow-up of 10 months), limiting the evaluation of long-term safety and the durability of the treatment effect.
• The prospective restriction of the trial exclusively to self-identified Black patients inherently limits the generalizability of these findings to other demographic populations.
• The primary endpoint was a complex, weighted composite score, which is less intuitive to interpret in clinical practice than traditional time-to-event outcomes.

Clinical Significance

The A-HeFT trial demonstrated that adding fixed-dose isosorbide dinitrate/hydralazine to standard neurohormonal blockade dramatically improves outcomes in self-identified Black patients with advanced heart failure. This profound survival benefit (43% mortality reduction) led to the FDA approval of BiDil specifically for this patient population, establishing it as a core component of guideline-directed medical therapy for African Americans with persistent symptomatic heart failure.

Historical Context

The rationale for A-HeFT stemmed from retrospective subgroup analyses of the earlier V-HeFT I and II trials, which suggested that African American patients experienced a unique survival benefit from the combination of isosorbide dinitrate and hydralazine, potentially due to underlying differences in nitric oxide bioavailability and oxidative stress. A-HeFT became a landmark—and widely debated—trial as the first prospective cardiovascular study designed to evaluate a therapy in a specific self-identified racial group, sparking significant scientific, ethical, and societal discussions regarding race-based medicine, pharmacogenomics, and the validity of using race as a proxy for genetic or physiological differences.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Hydralazine and isosorbide dinitrate (ISDN) target different aspects of hemodynamics. What are their respective primary sites of action (preload vs. afterload), and mechanistically, why are they given together rather than as monotherapy in heart failure?

Key Response

ISDN is primarily a venodilator that reduces preload by increasing venous capacitance, while hydralazine is a direct arterial vasodilator that reduces afterload. Together, they improve stroke volume and reduce cardiac work. Furthermore, hydralazine has potent antioxidant properties that mitigate the oxidative stress responsible for nitrate tolerance, allowing ISDN to remain effective during long-term therapy.

Resident
Resident

You are managing a 55-year-old African American male with NYHA class III HFrEF who is maximally titrated on standard guideline-directed medical therapy. You decide to add ISDN-hydralazine based on the A-HeFT trial. What are the most common adverse effects you should counsel him about, and what medication class is strictly contraindicated while he is on this therapy?

Key Response

The most common adverse effects requiring counseling are headache (driven by ISDN) and dizziness/hypotension. Hydralazine can also rarely cause drug-induced lupus erythematosus. Phosphodiesterase-5 (PDE-5) inhibitors (e.g., sildenafil) are strictly contraindicated due to the risk of profound, life-threatening hypotension when combined with nitrates.

Fellow
Fellow

The A-HeFT trial specifically enrolled self-identified Black patients. What is the proposed pathophysiological basis for the enhanced efficacy of ISDN-hydralazine in this demographic compared to others, and how does this relate to the concept of nitric oxide (NO) bioavailability?

Key Response

The working hypothesis is that Black patients with heart failure often have lower endothelial nitric oxide bioavailability and higher reactive oxygen species (ROS) burden compared to other demographics, leading to a NO-deficient state. ISDN acts as an exogenous NO donor, and hydralazine acts as an antioxidant to prevent NO degradation by ROS, specifically reversing this pathophysiologic deficit.

Attending
Attending

The introduction of BiDil (fixed-dose ISDN-hydralazine) sparked significant debate regarding race-based medicine, pill burden, and cost. How do you balance the robust mortality benefit seen in A-HeFT with the practical complexities of prescribing a therapy marketed for a specific racial group, especially when managing patients with mixed heritage or those struggling with polypharmacy?

Key Response

This addresses the real-world complexity of applying A-HeFT. Race is a social construct, making it biologically imprecise to limit life-saving therapy based strictly on self-identification. Attendings must often decide whether to trial it in non-Black patients who remain symptomatic despite optimal medical therapy. Additionally, the TID dosing of ISDN-hydralazine creates a high pill burden, meaning shared decision-making regarding adherence and financial toxicity is crucial for achieving the outcomes seen in the trial.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

A-HeFT utilized a novel primary composite scoring system that incorporated all-cause mortality, first hospitalization for heart failure, and change in quality of life. What are the methodological strengths and weaknesses of using this type of hierarchical composite endpoint rather than a traditional time-to-first-event analysis?

Key Response

A hierarchical composite score (similar to the Finkelstein-Schoenfeld method) increases statistical power and appropriately weights mortality over hospitalization and quality of life, solving the issue of standard time-to-first-event composites where a hospitalization could mask a later death. However, its primary weakness is that it complicates the clinical interpretation of the magnitude of benefit and makes direct cross-trial comparisons difficult.

Journal Editor
Journal Editor

The A-HeFT trial was terminated early by the Data and Safety Monitoring Board due to a significant mortality benefit. As an editor, how do you evaluate the potential for exaggerated effect sizes in trials stopped early for benefit, and how does the lack of a non-Black comparator arm impact the external validity and editorial framing of the findings?

Key Response

Trials stopped early for benefit are susceptible to the 'winner's curse,' often overestimating the true treatment effect due to random high fluctuations at the time of interim analysis. Furthermore, restricting the trial exclusively to Black patients removes the ability to prospectively compare the effect size against other groups, leaving it ambiguous whether the therapy is uniquely effective in this population or simply effective across the board but only tested in one group.

Guideline Committee
Guideline Committee

Based on the A-HeFT trial and subsequent data, how do the current ACC/AHA/HFSA guidelines recommend the use of ISDN-hydralazine in HFrEF? Specifically, what is the Class of Recommendation for self-identified African American patients, and in what alternative clinical scenario is this combination recommended for non-African American patients?

Key Response

Current ACC/AHA/HFSA guidelines give a Class 1 recommendation for adding ISDN-hydralazine to optimal medical therapy in self-identified African American patients with NYHA class III-IV HFrEF to reduce morbidity and mortality. Additionally, it holds a Class 2a/2b recommendation for any patient (regardless of race) with current or prior symptomatic HFrEF who cannot tolerate an ACEi, ARB, or ARNI due to severe renal impairment or hyperkalemia.

Clinical Landscape

Noteworthy Related Trials

1986

V-HeFT I

n = 642 · NEJM

Tested

Hydralazine and Isosorbide Dinitrate

Population

Men with chronic heart failure

Comparator

Placebo or Prazosin

Endpoint

All-cause mortality

Key result: The combination of hydralazine and isosorbide dinitrate resulted in a borderline significant reduction in mortality compared to placebo.
1991

V-HeFT II

n = 804 · NEJM

Tested

Enalapril

Population

Men with chronic heart failure taking digoxin and diuretics

Comparator

Hydralazine and Isosorbide Dinitrate

Endpoint

All-cause mortality

Key result: Enalapril significantly reduced mortality compared to the hydralazine and isosorbide dinitrate combination overall.
1991

SOLVD Treatment Trial

n = 2569 · NEJM

Tested

Enalapril

Population

Patients with heart failure and LVEF of 35 percent or less

Comparator

Placebo

Endpoint

All-cause mortality

Key result: Enalapril significantly reduced all-cause mortality and heart failure hospitalizations.

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