What are the most effective medicines with the lowest Number Needed to Treat (NNT)?

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Last updated: May 22, 2025View editorial policy

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From the Guidelines

The most effective medicines with the lowest number needed to treat (NNT) for heart failure include mineralocorticoid receptor antagonists (MRA) such as spironolactone, with an NNT of 9 for reducing mortality in patients with severe heart failure, as demonstrated in the RALES trial 1. These medications have been shown to provide significant benefits in reducing morbidity and mortality in patients with heart failure, particularly those with reduced ejection fraction. The use of MRAs, such as spironolactone and eplerenone, has been associated with a reduced risk of death and hospitalization due to heart failure, with an NNT of 9 for spironolactone in the RALES trial 1. Other effective treatments for heart failure include ACE inhibitors, beta-blockers, and ARBs, which have been shown to reduce morbidity and mortality in patients with heart failure, although their NNT values may be higher than those of MRAs. For example, the EMPHASIS-HF trial demonstrated that eplerenone reduced the risk of cardiovascular death or heart failure hospitalization by 37% in patients with systolic heart failure and mild symptoms 1. When implementing these treatments, it's essential to consider patient-specific factors, including comorbidities, contraindications, and potential adverse effects, to optimize the benefit-risk ratio. Some key evidence-based doses of disease-modifying drugs used in heart failure include:

  • MRA: spironolactone 25-50 mg once daily, eplerenone 25-50 mg once daily
  • ACE inhibitors: captopril 6.25-50 mg three times daily, enalapril 2.5-20 mg twice daily
  • Beta-blockers: bisoprolol 1.25-10 mg once daily, carvedilol 3.125-50 mg twice daily
  • ARBs: candesartan 4-32 mg once daily, valsartan 40-160 mg twice daily These medications should be used in accordance with established guidelines and patient-specific factors to achieve optimal outcomes. In contrast, the provided evidence on treatments for alcohol abstinence 1 is not directly relevant to the question of effective medicines for heart failure, and therefore, is not considered in this recommendation.

From the Research

Most Effective Medicines

The most effective medicines with the lowest number needed to treat (NNT) can be determined by analyzing various studies on antihypertensive and cholesterol-lowering therapies.

  • The study 2 found that lisinopril, losartan, and valsartan, alone or in combination with hydrochlorothiazide, were equally effective in reducing blood pressure and improving arterial stiffness in patients with essential hypertension.
  • Another study 3 demonstrated that the combination of lisinopril, amlodipine, and rosuvastatin was well-tolerated and effective in patients with mild to moderate hypertension and hypercholesterolemia.
  • The study 4 showed that the fixed-dose combination of amlodipine and atorvastatin led to a lower risk of composite cardiovascular outcomes compared to the free combination or a similar fixed-dose combination with a lower dose of atorvastatin.

Number Needed to Treat (NNT)

The concept of NNT is crucial in determining the effectiveness of a treatment, as it provides a measure of the number of patients needed to be treated to achieve a beneficial outcome 5, 6.

  • The NNT is calculated as the inverse of the absolute risk reduction and is most meaningful when reported within a confidence interval and based on high-validity clinical trials such as randomized controlled trials and meta-analyses.
  • The study 6 highlights the importance of NNT in comparing the relative effectiveness of different therapeutic interventions and provides examples of NNTs from the dental and medical literature.

Low NNT Medicines

Medicines with a low NNT are considered more effective, as they require fewer patients to be treated to achieve a beneficial outcome.

  • The combination of lisinopril, amlodipine, and rosuvastatin 3 and the fixed-dose combination of amlodipine and atorvastatin 4 have been shown to be effective in reducing cardiovascular risk factors and may have a low NNT.
  • However, the exact NNT values for these medicines are not provided in the studies, and further research is needed to determine their NNT values.

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Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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