Cheaper muscular dystrophy drug may be as effective as expensive alternative

Written by Ilana Landau, Editor

The results of a new, comparative effectiveness clinical trial demonstrate that spironolactone may be as effective as eplerenone at preserving cardiovascular function in Duchenne muscular dystrophy patients, whilst being more cost effective too.

Spironolactone and eplerenone are two drugs often prescribed for the management of hypertension and heart failure. In a new study, researchers from the Ohio State University College of Medicine (OH, USA) have directly compared the effectiveness of spironolactone with eplerenone at protecting cardiovascular function in Duchenne muscular dystrophy patients. The results of the study demonstrate that spironolactone is as effective at conserving normal heart function as eplerenone, whilst being significantly cheaper.

The results of the multi-center, double-blind trial, published in the Journal of the American Heart Association, concern 52 boys with Duchene muscular dystrophy aged 12—18. All trial enrollees received cardiac imaging prior to study commencement and had preserved cardiac function at the start of the trial. Participants were randomly assigned to receive either 50 mg of eplerenone or spironolactone, once a day, for 1 year.

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At the end of the 1-year study, researchers observed no significant changes in any patients’ cardiac, kidney or lung functions.

Lead study researcher Subha Raman, a Cardiologist and Professor at Ohio State, commented: “Even though it works well, the concern with eplerenone is that it’s more costly and less available worldwide. Spironolactone is more commonly available and can cost about 75% less, making it more accessible to everyone who could benefit.”


What is Duchene muscular dystrophy?

Duchene muscular dystrophy is a genetic disorder, most common amongst boys, in which there is a lack of production of dystrophin protein. Dystrophin is important for stabilizing muscle cells. As a result, patients experience progressive muscle weakening.

How does spironolactone work?

Spironolactone is a competitive antagonist of aldosterone. It works by preventing the uptake of sodium in the distal tubules in the kidneys, thus acting to increase water excretion. 

How does eplerenone work?

Epleronone is also an antagonist of aldosterone receptors. Epleronone therefore increases water output by the kidneys by preventing aldosterone-mediated sodium reuptake.