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TOPCAT

"Spironolactone for Heart Failure with Preserved Ejection Fraction (TOPCAT Study)". The New England Journal of Medicine. 2014;370:1383-1392. PubMed

Links to original sources: Wiki Journal Post Full Journal Article

Clinical Question


Does treatment with spironolactone improve the prognosis for patients with heart failure and a preserved left ventricular ejection fraction?

Bottom Line


In patients with heart failure and a preserved ejection fraction, treatment with spironolactone did not significantly reduce the composite primary outcome of death from cardiovascular causes, aborted cardiac arrest, or hospitalization for heart failure management.

Major Points


Heart failure with preserved ejection fraction (HFpEF) is a common condition with no specifically approved treatments to improve patient outcomes. While mineralocorticoid-receptor antagonists have proven effective in reducing mortality and hospitalizations for heart failure in patients with reduced ejection fraction, little evidence has been available for HFpEF. The TOPCAT trial sought to fill this gap by evaluating the effects of spironolactone on clinical outcomes in HFpEF.

Guidelines


Current guidelines offer no specific recommendations for the management of heart failure in patients with preserved ejection fraction, except for attention to coexisting conditions.

Design


- Multicenter, international, randomized, double-blind, placebo-controlled trial - N=3,445 patients with symptomatic heart failure and left ventricular ejection fraction ≥45% - Spironolactone (15 to 45 mg daily) vs placebo - Mean follow-up: 3.3 years

Population


- Inclusion Criteria: Patients ≥50 years of age, with symptomatic heart failure, left ventricular ejection fraction ≥45%, hospitalization within the previous 12 months for heart failure management, or elevated natriuretic peptide levels. - Exclusion Criteria: Severe systemic illness, severe renal dysfunction, various coexisting conditions and medications, and recent acute events.

Interventions


- Initial dose: 15 mg spironolactone or placebo once daily, increased to a maximum of 45 mg daily during the first 4 months after randomization. - Subsequent dose adjustments as required with frequent monitoring for serum potassium and creatinine levels.

Outcomes


- Primary Outcome: Composite of death from cardiovascular causes, aborted cardiac arrest, or hospitalization for heart failure management. - Secondary Outcome: Death from any cause, hospitalization for any cause, hyperkalemia, hypokalemia, elevated serum creatinine level.

Criticisms


- A significant proportion of participants discontinued the study drug, primarily due to elevations in serum creatinine and potassium levels. - There were marked regional differences in event rates, which may reflect heterogeneity in coexisting conditions and practice patterns.

Funding


- The National Heart, Lung, and Blood Institute; contract HHSN268200425207C

Further Reading


- Full TOPCAT trial publication in The New England Journal of Medicine