JAMA September 17, 2019

Association of Change in N-Terminal Pro-B-Type Natriuretic Peptide Following Initiation of Sacubitril-Valsartan Treatment With Cardiac Structure and Function in Patients With Heart Failure With Reduced Ejection Fraction

James L Januzzi Jr, Margaret F Prescott, Javed Butler, G Michael Felker, Alan S Maisel, Ileana L Piña, Scott D Solomon, et al. (PROVE-HF Investigators)

Bottom Line

In patients with HFrEF, the initiation of sacubitril-valsartan led to rapid reductions in NT-proBNP that correlated with significant reverse cardiac remodeling and improvements in left ventricular ejection fraction at 12 months.

Key Findings

1. Among 794 enrolled patients, median NT-proBNP concentrations significantly decreased from 816 pg/mL at baseline to 455 pg/mL at 12 months (P < .001).
2. At 12 months, the change in log2-NT-proBNP concentration was significantly correlated with improvements in left ventricular ejection fraction (LVEF) (r = -0.381).
3. Reductions in NT-proBNP also significantly correlated with structural improvements, including left ventricular end-diastolic volume index (LVEDVI) (r = 0.320) and left ventricular end-systolic volume index (LVESVI) (r = 0.405).
4. Overall, median LVEF demonstrated a marked absolute increase from 28.2% at baseline to 37.8% at 12 months (an absolute difference of 9.4%; P < .001).
5. Significant structural reductions were observed in LVEDVI (median 86.9 to 74.1 mL/m2) and LVESVI (median 61.7 to 45.5 mL/m2) over 12 months.

Study Design

Design
Single-Arm Trial
Open-Label
Sample
794
Patients
Duration
12 mo
Median
Setting
Multicenter, US
Population Adults with symptomatic heart failure with reduced ejection fraction (HFrEF) and an LVEF ≤ 40%.
Intervention Initiation and titration of sacubitril-valsartan over a 12-month period.
Comparator None (baseline measurements served as the intra-patient comparator).
Outcome Correlation between changes in log2-NT-proBNP concentrations and echocardiographic measures of cardiac remodeling (LVEF, LVEDVI, LVESVI, LAVI, and E/e' ratio) at 12 months.

Study Limitations

Open-label, single-arm study design lacks a randomized control or active comparator group, making it impossible to definitively attribute all remodeling exclusively to the drug versus background therapy optimization.
Not all patients completed the study; approximately 18% of the initial 794 patients did not finish the 12-month follow-up.
Some patients had missing echocardiographic data at the 6- or 12-month follow-up time points.
The observed statistical correlations between NT-proBNP reductions and echocardiographic changes do not definitively prove causality.

Clinical Significance

PROVE-HF provides a crucial mechanistic link explaining the profound mortality and morbidity benefits of sacubitril/valsartan observed in the PARADIGM-HF trial. By demonstrating that ARNI therapy drives robust reverse cardiac remodeling (an absolute LVEF increase of nearly 10%) that mirrors declines in NT-proBNP, the study validates serial NT-proBNP measurement as a reliable clinical tool for tracking therapeutic response and physiologic improvement in patients with HFrEF.

Historical Context

The 2014 PARADIGM-HF trial was a landmark study that established sacubitril/valsartan as superior to enalapril in reducing death and heart failure hospitalization. However, the exact physiological mechanisms underlying this marked benefit were not fully elucidated. PROVE-HF was subsequently designed to determine if the clinical efficacy of angiotensin receptor-neprilysin inhibition is mediated through reverse cardiac remodeling, and whether reductions in natriuretic peptides directly correlate with these structural improvements.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Why is NT-proBNP, rather than BNP, the preferred biomarker for monitoring treatment response and reverse remodeling in patients initiated on sacubitril-valsartan?

Key Response

Sacubitril inhibits neprilysin, an enzyme that degrades BNP. Therefore, BNP levels will artificially rise upon initiation of an ARNI, independent of heart failure status. NT-proBNP is not a substrate for neprilysin, making it an accurate reflection of true ventricular wall stress and subsequent reverse remodeling.

Resident
Resident

Based on the PROVE-HF findings, how should the rapid decline in NT-proBNP following ARNI initiation influence your timeline for ordering a follow-up echocardiogram to assess for ICD indication?

Key Response

The study showed that NT-proBNP drops rapidly within 1 to 2 weeks, but structural reverse remodeling and LVEF improvement take several months, peaking around 12 months. Therefore, a rapid drop in NT-proBNP should reassure the clinician that the therapy is working, but echo reassessment for device therapy should be delayed for at least 3 to 6 months on optimal medical therapy, as LVEF is likely to continue improving.

Fellow
Fellow

PROVE-HF demonstrated a correlation between early NT-proBNP reduction and late reverse remodeling. What is the physiological significance of the subset of patients who achieve reverse remodeling without a proportional drop in NT-proBNP, and how might their distinct pathophysiology dictate different advanced HF therapies?

Key Response

While the median trend showed correlation, inter-individual variability exists. Patients lacking NT-proBNP reduction despite ARNI therapy may have isolated predominant right ventricular dysfunction, distinct fibrotic phenotypes, or alternative clearance mechanisms masking the biomarker's utility. Recognizing this uncoupling is critical to prevent prematurely withdrawing a beneficial therapy or inappropriately accelerating a patient toward transplant or LVAD evaluation.

Attending
Attending

Many patients in this study experienced an LVEF increase to over 40 percent, transitioning them to HFimpEF. As an attending, how do you use the mechanistic findings of PROVE-HF combined with the TRED-HF trial to counsel patients who request to stop their ARNI once their ejection fraction has 'normalized'?

Key Response

PROVE-HF proves that ARNI drives profound structural reverse remodeling. However, TRED-HF showed that withdrawing therapy in recovered dilated cardiomyopathy leads to rapid relapse. The attending teaching point is that ARNI-induced remodeling is a state of remission, not a definitive cure; the biochemical environment evidenced by NT-proBNP control must be maintained to sustain the structural gains.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

PROVE-HF was an open-label, single-arm study without a concurrent control group. Statistically, how might 'regression to the mean' and 'survivor bias' confound the magnitude of the observed association between NT-proBNP changes and echocardiographic reverse remodeling?

Key Response

Without a randomized control group, it is statistically challenging to isolate the specific effect of sacubitril-valsartan from the natural trajectory of the disease or the optimization of concomitant baseline background therapies. Furthermore, patients who survived and tolerated the medication to 12 months to get the final echo represent a survivor cohort, potentially overestimating the overall population-level remodeling benefit.

Journal Editor
Journal Editor

As an editor evaluating the PROVE-HF manuscript, what major threats to internal validity arise from the 12-month echocardiographic follow-up attrition rate, and how does the lack of a blinded control arm affect the causal attribution of reverse remodeling exclusively to the ARNI?

Key Response

A tough reviewer would flag the missing data at 12 months due to dropouts, intolerance, or death which inherently biases the paired echo analysis toward responders. Furthermore, since it is an open-label study, concurrent uptitration of other GDMT could independently drive reverse remodeling, making it difficult to firmly attribute the entire LVEF improvement strictly to the sacubitril-valsartan initiation.

Guideline Committee
Guideline Committee

Current heart failure guidelines recommend waiting 3 to 6 months after optimizing GDMT before reassessing LVEF for primary prevention ICD placement. How does the temporal trajectory of reverse remodeling demonstrated in PROVE-HF validate or challenge this specific guideline recommendation?

Key Response

PROVE-HF strongly supports and extends this guideline. It demonstrated that while biochemical response is rapid, structural remodeling is continuous and may take up to 12 months. This provides robust evidence for the guideline committee to emphasize delaying permanent device implantation in patients recently initiated on an ARNI, as premature implantation might occur in patients destined for profound delayed recovery.

Clinical Landscape

Noteworthy Related Trials

2014

PARADIGM-HF

n = 8,399 · NEJM

Tested

Sacubitril-valsartan 200 mg twice daily

Population

Patients with chronic HFrEF

Comparator

Enalapril 10 mg twice daily

Endpoint

Composite of cardiovascular death or heart failure hospitalization

Key result: Sacubitril-valsartan significantly reduced the risk of cardiovascular death or heart failure hospitalization by 20% compared to enalapril.
2019

PIONEER-HF

n = 881 · NEJM

Tested

Sacubitril-valsartan initiated in-hospital

Population

Patients hospitalized for acute decompensated heart failure with HFrEF

Comparator

Enalapril

Endpoint

Time-averaged proportional change in NT-proBNP from baseline through weeks 4 and 8

Key result: Initiation of sacubitril-valsartan led to a significantly greater reduction in NT-proBNP concentration than enalapril therapy.
2019

EVALUATE-HF

n = 464 · JAMA

Tested

Sacubitril-valsartan

Population

Patients with HFrEF

Comparator

Enalapril

Endpoint

Change in aortic characteristic impedance at 12 weeks

Key result: No significant difference in aortic impedance, but sacubitril-valsartan showed improvements in several echocardiographic measures of cardiac remodeling.

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